Sacituzumab govitecan

Sacituzumab govitecan-hziy is a Trop-2-directed antibody-drug conjugate. Sacituzumab is a humanized antibody that recognizes Trop-2. The small molecule, SN-38, is a topoisomerase I inhibitor, which is covalently attached to the antibody by a linker. Pharmacology data suggest that sacituzumab govitecan-hziy binds to Trop-2-expressing cancer cells and is internalized with the subsequent release of SN-38 via hydrolysis of the linker. SN-38 interacts with topoisomerase I and prevents re-ligation of topoisomerase I-induced single strand breaks. The resulting DNA damage leads to apoptosis and cell death. Sacituzumab govitecan-hziy decreased tumor growth in mouse xenograft models of triple-negative breast cancer


triple-negative breast carcinoma

biolink:Disease biolink:DiseaseOrPhenotypicFeature biolink:ThingWithTaxon biolink:BiologicalEntity biolink:NamedThing biolink:Entity


Capmatinib

Capmatinib is a kinase inhibitor indicated for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation that leads to mesenchymal-epithelial transition (MET) exon 14 skipping as detected by an FDA-approved test.


Selpercatinib

Selpercatinib is a kinase inhibitor. Selpercatinib inhibited wild-type RET and multiple mutated RET isoforms as well as VEGFR1 and VEGFR3 with IC50 values ranging from 0.92 nM to 67.8 nM. In other enzyme assays, selpercatinib also inhibited FGFR 1, 2, and 3 at higher concentrations that were still clinically achievable. In cellular assays, selpercatinib inhibited RET at approximately 60-fold lower concentrations than FGFR1 and 2 and approximately 8-fold lower concentration than VEGFR3


Ripretinib

Ripretinib is a tyrosine kinase inhibitor that inhibits KIT proto-oncogene receptor tyrosine kinase (KIT) and platelet derived growth factor receptor A (PDGFRA) kinase, including wild type, primary, and secondary mutations. Ripretinib also inhibits other kinases in vitro, such as PDGFRB, TIE2, VEGFR2, and BRAF


Fluoroestradiol F-18

Fluoroestradiol F 18 binds ER and it is a radioactive diagnostic agent indicated for positron emission tomography (PET) imaging. The following binding affinity: Kd = 0.13 ± 0.02 nM, Bmax = 1901 ± 89 fmol/mg, and IC50 = 0.085 nM, was determined in an ER-positive human breast cancer cell line (MCF-7). Fluoroestradiol F 18 is indicated for characterization of estrogen receptor status of known or suspected metastatic lesions in patients with confirmed ER-positive breast cancer.


Positron-Emission Tomography

biolink:Procedure biolink:ActivityAndBehavior biolink:NamedThing biolink:Entity biolink:Occurrent biolink:PhysicalEssenceOrOccurrent


flortaucipir (18F)

Flortaucipir F 18 is a radioactive diagnostic agent indicated for positron emission tomography (PET) imaging of the brain to estimate the density and distribution of aggregated tau neurofibrillary tangles (NFTs) in adult patients with cognitive impairment who are being evaluated for Alzheimer’s disease (AD). It binds to aggregated tau protein. In the brains of patients with AD, tau aggregates combine to form NFTs, one of two components required for the neuropathological diagnosis of AD. In vitro, flortaucipir F 18 binds to paired helical filament (PHF) tau purified from brain homogenates of donors with AD. The dissociation constant (Kd) of flortaucipir F 18 binding to PHFs is 0.57 nM. In vivo, flortaucipir F 18 is differentially retained in neocortical areas that contain aggregated tau. In vitro, tritiated flortaucipir has been reported to bind with low nanomolar affinity to monoamine oxidase-A and monoamine oxidase-B, which could contribute to off target binding


INEBILIZUMAB

Inebilizumab-cdon is a CD19-directed humanized afucosylated IgG1 monoclonal antibody produced by recombinant DNA technology in Chinese hamster ovary (CHO) cell suspension culture. The precise mechanism by which inebilizumab-cdon exerts its therapeutic effects in NMOSD is unknown but is presumed to involve binding to CD19, a cell surface antigen presents on pre-B and mature B lymphocytes. Following cell surface binding to B lymphocytes, inebilizumab-cdon results in antibody-dependent cellular cytolysis (ADCC).


lurbinectedin

Lurbinectedin is an alkylating drug that binds guanine residues in the minor groove of DNA, forming adducts and resulting in a bending of the DNA helix towards the major groove. Adduct formation triggers a cascade of events that can affect the subsequent activity of DNA binding proteins, including some transcription factors, and DNA repair pathways, resulting in perturbation of the cell cycle and eventual cell death.


Remimazolam

Remimazolam is a benzodiazepine and it binds to brain benzodiazepine sites (gamma amino butyric acid type A [GABAA] receptors), while its carboxylic acid metabolite (CNS7054) has a 300 times lower affinity for the receptor. Remimazolam, like other benzodiazepines, did not show clear selectivity between subtypes of the GABAA receptor.


Cedazuridine

Cedazuridine inhibits cytidine deaminase (CDA), an enzyme that catalyzes the degradation of cytidine, including the cytidine analog decitabine (a nucleoside metabolic inhibitor). Because high levels of CDA in the gastrointestinal tract and liver degrade decitabine and limit its oral bioavailability, administration of cedazuridine with decitabine increases systemic exposure of decitabine. Inqovi is a combination of decitabine and cedazuridine for treatment of adult patients with myelodysplastic syndromes (MDS).


Abametapir

Abametapir is a metalloproteinase inhibitor. Metalloproteinases have a role in physiological processes critical to egg development and survival of lice.


TAFASITAMAB

Tafasitamab-cxix is an Fc-modified monoclonal antibody that binds to CD19 antigen expressed on the surface of pre-B and mature B lymphocytes and on several B-cell malignancies, including diffuse large B-cell lymphoma (DLBCL). Upon binding to CD19, tafasitamab-cxix mediates B-cell lysis through apoptosis and immune effector mechanisms, including antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP).


Diffuse large B-cell lymphoma refractory


belantamab mafodotin

Belantamab mafodotin-blmf is an antibody-drug conjugate (ADC). The antibody component is an afucosylated IgG1 directed against BCMA, a protein expressed on normal B lymphocytes and multiple myeloma cells. The small molecule component is MMAF, a microtubule inhibitor. Upon binding to BCMA, belantamab mafodotin-blmf is internalized followed by release of MMAF via proteolytic cleavage. The released MMAF intracellularly disrupts the microtubule network, leading to cell cycle arrest and apoptosis.


oliceridine

Oliceridine is a full opioid agonist and is relatively selective for the mu-opioid receptor. The principal therapeutic action of oliceridine is analgesia. Like all full opioid agonists, there is no ceiling effect to analgesia for oliceridine. Clinically, dosage is titrated to provide adequate analgesia and may be limited by adverse reactions, including respiratory, and CNS depression. The precise mechanism of the analgesic action is unknown. However, specific CNS opioid receptors for endogenous compounds with opioid-like activity have been identified throughout the brain and spinal cord and are thought to play a role in the analgesic effects of this drug.


Severe pain

biolink:PhenotypicFeature biolink:DiseaseOrPhenotypicFeature biolink:ThingWithTaxon biolink:BiologicalEntity biolink:NamedThing biolink:Entity


Risdiplam

Risdiplam is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat patients with spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, risdiplam was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein in the brain.


viltolarsen

Viltolarsen is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients. It is designed to bind to exon 53 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping.


Cepharanthine

The mechanism of action of cepharanthine is multifactorial. The drug exerts membrane effects (modulation of efflux pumps, membrane rigidification) as well as different intracellular and nuclear effects. Cepharanthine interferes with several metabolic axes, primarily with the AMP-activated protein kinase (AMPK) and NFkappaB signaling pathways. In particular, the anti-inflammatory effects of cepharanthine rely on AMPK activation and NFkappaB inhibition.


Baloxavir marboxil

Baloxavir marboxil is a prodrug that is converted by hydrolysis to baloxavir, the active form that exerts anti-influenza virus activity. Baloxavir inhibits the endonuclease activity of the polymerase acidic (PA) protein, an influenza virus-specific enzyme in the viral RNA polymerase complex required for viral gene transcription, resulting in inhibition of influenza virus replication. The 50% inhibitory concentration (IC50) of baloxavir was 1.4 to 3.1 nM (n=4) for influenza A viruses and 4.5 to 8.9 nM (n=3) for influenza B viruses in a PA endonuclease assay. Viruses with reduced susceptibility to baloxavir have amino acid substitutions in the PA protein. It was investigated as a potential treatment during the early stages of the COVID-19 pandemic but in vitro antiviral activity against SARS-CoV-2 was not confirmed.


Chloroquine

The prototypical antimalarial agent with a mechanism that is not well understood. It has also been used to treat rheumatoid arthritis, systemic lupus erythematosus, and in the systemic therapy of amebic liver abscesses. In March 2020, FDA issued an emergency use authorization (EUA) for hydroxychloroquine and chloroquine in the treatment of COVID-10. EUA was revoked in June 2020. Data from various published randomized, controlled clinical trials and retrospective, cohort studies have not substantiated  initial reports of efficacy of 4-aminoquinoline antimalarials for treatment of COVID-19.


Favipiravir

A pyrazinecarboxamide derivative with activity against RNA viruses. Favipiravir is converted to the ribofuranosyltriphosphate derivative by host enzymes and selectively inhibits the influenza viral RNA-dependent RNA polymerase. Several studies reported the use of favipiravir for the treatment of COVID-19 but additional data are needed to substantiate the initial reports. Given the lack of pharmacokinetic and safety data for the high favipiravir dosages proposed for treatment of COVID-19, the drug should be used with caution at such dosages.


Lopinavir

An HIV protease inhibitor used in a fixed-dose combination with RITONAVIR. It is also an inhibitor of CYTOCHROME P-450 CYP3A. There was some evidence of in vitro activity against SARS-CoV-2, but no clinical trial data was found to support use in the treatment of COVID-19.


Darunavir

An HIV PROTEASE INHIBITOR that is used in the treatment of AIDS and HIV INFECTIONS. Due to the emergence of ANTIVIRAL DRUG RESISTANCE when used alone, it is administered in combination with other ANTI-HIV AGENTS. There was some evidence of in vitro activity against SARS-CoV-2, but no clinical trial data was found to support use in the treatment of COVID-19.


Hydroxychloroquine

A chemotherapeutic agent that acts against erythrocytic forms of malarial parasites. Hydroxychloroquine appears to concentrate in food vacuoles of affected protozoa. It inhibits plasmodial heme polymerase. (From Gilman et al., Goodman and Gilman's The Pharmacological Basis of Therapeutics, 9th ed, p970). In March 2020, FDA issued an emergency use authorization (EUA) for hydroxychloroquine and chloroquine in the treatment of COVID-10. EUA was revoked in June 2020. Data from various published randomized, controlled clinical trials and retrospective, cohort studies have not substantiated  initial reports of efficacy of 4-aminoquinoline antimalarials for treatment of COVID-19.


Oseltamivir

Oseltamivir phosphate is an ethyl ester prodrug requiring ester hydrolysis for conversion to the active form, oseltamivir carboxylate. Oseltamivir carboxylate is an inhibitor of influenza virus neuraminidase affecting release of viral particles. The median IC50 values of oseltamivir against influenza A/H1N1, influenza A/H3N2, and influenza B clinical isolates were 2.5 nM (range 0.93-4.16 nM, N=74), 0.96 nM (range 0.13 - 7.95 nM, N=774), and 60 nM (20-285 nM, N=256), respectively, in a neuraminidase assay with a fluorescently labeled MUNANA substrate. Although oseltamivir was suggested as a potential treatment and included in various antiviral regimens used during the early stages of the COVID-19 pandemic, the drug does not appear to have in vitro  activity against SARS-CoV-2 and there are no data to support the use of oseltamivir or other neuraminidase inhibitors in the treatment of COVID-19.


Remdesivir

Remdesivir is an investigational nucleotide analog with broad-spectrum antiviral activity. Remdesivir has demonstrated in vitro and in vivo activity in animal models against the viral pathogens MERS and SARS, which are also coronaviruses and are structurally similar to COVID-19. The limited preclinical data on remdesivir in MERS and SARS indicate that remdesivir may have potential activity against COVID-19. The only direct-acting antiviral (DAA) currently approved by FDA for the treatment of COVID-19 in certain populations.


Arbidol

an antiviral agent. Although data is limited, in vitro activity against SARS-CoV-1 and SARS-CoV-2 has been reported. The drug has been included in COVID-19 treatment guidelines used in China and Russia.


EMAPALUMAB

Emapalumab-lzsg is a monoclonal antibody that binds to and neutralizes interferon gamma  (IFNgamma). Nonclinical data suggest that IFNgamma plays a pivotal role in the pathogenesis of HLH by being hypersecreted.


Ascorbic Acid

A six carbon compound related to glucose. It is found naturally in citrus fruits and many vegetables. Ascorbic acid is an essential nutrient in human diets, and necessary to maintain connective tissue and bone. Its biologically active form, vitamin C, functions as a reducing agent and coenzyme in several metabolic pathways. Vitamin C is considered an antioxidant. NIH COVID-19 Treatment Guidelines Panel states that there are insufficient data to recommend either for or against use of ascorbic acid for the treatment of COVID-19 in critically or noncritically ill patients.


Azithromycin

A semi-synthetic macrolide antibiotic structurally related to ERYTHROMYCIN. It has been used in the treatment of Mycobacterium avium intracellulare infections, toxoplasmosis, and cryptosporidiosis. Some evidence of in vitro activity against SARS-CoV-2 in infected Vero E6 and Caco-2 cells has been reported but clinical importance it is unclear. Only limited information available regarding the frequency and microbiology of bacterial pulmonary coinfections or superinfections in pacients with COVID-19.


Methylprednisolone

A PREDNISOLONE derivative with similar anti-inflammatory action.


Ruxolitinib

a kinase inhibitor, inhibits Janus Associated Kinases (JAKs) JAK1 and JAK2 which mediate the signaling of a number of cytokines and growth factors that are important for hematopoiesis and immune function. Although some small studies have suggested possibility of benefit from ruxolitinib in patients with COVID-19, it is not recommended the use of JAK inhibitors other than baricitinib for the treatment of COVID-19.


Sirolimus

A macrolide compound obtained from Streptomyces hygroscopicus that acts by selectively blocking the transcriptional activation of cytokines thereby inhibiting cytokine production. It is bioactive only when bound to IMMUNOPHILINS. Sirolimus is a potent immunosuppressant and possesses both antifungal and antineoplastic properties. Limited data describing efficacy in patients with COVID-19 is available. However, its efficacy and safety in the treatment of COVID-19 was not established.


Sodium heparin

A highly acidic mucopolysaccharide formed of equal parts of sulfated D-glucosamine and D-glucuronic acid with sulfaminic bridges. The molecular weight ranges from six to twenty thousand. Heparin occurs in and is obtained from liver, lung, mast cells, etc., of vertebrates. Its function is unknown, but it is used to prevent blood clotting in vivo and vitro, in the form of many different salts.


Nitazoxanide

a 5-nitrothiazolyl derivative used for a broad range of intestinal parasitic infections including CRYPTOSPORIDIUM and GIARDIA; it is a redox-active nitrothiazolyl-salicylamide prodrug; structure given in first source. It was investigated as a potential treatment for COVID-19 based on its broad antiviral activity, including in vitro activity against SARS-CoV-2 and MERS-CoV;  however, there are no data to support the use of nitazoxanide in the treatment of COVID-19.


Ribavirin

A nucleoside antimetabolite antiviral agent that blocks nucleic acid synthesis and is used against both RNA and DNA viruses.


Drostanolone propionate

synthetic anabolic androgenic steroid used to lower plasma cholesterol & as antineoplastic agent in advanced breast neoplasms; major descriptor (66-86); on-line search ANDROSTANOLS (80-86); ANDROSTANES (68-86); INDEX MEDICUS search DROMOSTANOLONE (66-86); RN given refers to (2alpha,5alpha,17beta)-isomer


Benzbromarone

Uricosuric that acts by increasing uric acid clearance. It is used in the treatment of gout.


Tafenoquine

Tafenoquine, an 8-aminoquinoline antimalarial, is active against all the stages of Plasmodium species that include the hypnozoite (dormant stage) in the liver. Studies in vitro with the erythrocytic forms of Plasmodium falciparum suggest that tafenoquine may exert its effect by inhibiting hematin polymerization and inducing apoptotic like death of the parasite. In addition to its effect on the parasite, tafenoquine causes red blood cell shrinkage in vitro. The molecular target of tafenoquine is not known.


mecasermin rinfabate

Mecasermin rinfabate [rDNA origin] injection is an aqueous solution for injection containing a binary protein complex of human insulin-like growth factor-1 (rhIGF-1) and human insulin-like growth factor-binding protein-3 (rhIGFBP-3), both produced by recombinant DNA technology. The primary pharmacologic effect of IGF-1 in children is the promotion of linear growth. Secondary pharmacologic actions of IGF-1 include other anabolic effects, insulin sensitization, and insulin-like effects. There are no known direct growth-promoting effects of IGFBP-3. The primary effect of IGFBP-3 in the mecasermin rinfabate complex is the modulation of IGF-1 action.


Oxaceprol

Oxaceprol, is an atypical inhibitor of inflammation which helps in reduction of leukocyte adherence for joint diseases. This medicine is used for osteoarthritis or degenerative joint disorders and inflammation of connective tissues.


IBALIZUMAB

Ibalizumab-uiyk, a recombinant humanized monoclonal antibody, blocks HIV-1 from infecting CD4+ T cells by binding to domain 2 of CD4 and interfering with post-attachment steps required for the entry of HIV-1 virus particles into host cells and preventing the viral transmission that occurs via cell-cell fusion


Fostamatinib

Fostamatinib is a tyrosine kinase inhibitor with demonstrated activity against spleen tyrosine kinase (SYK). The major metabolite of fostamatinib, R406, inhibits signal transduction of Fc-activating receptors and B-cell receptor. The fostamatinib metabolite R406 reduces antibody-mediated destruction of platelets.


thrombocytopenia due to immune destruction


tildrakizumab

Tildrakizumab is a humanized IgG1/k monoclonal antibody that selectively binds to the p19 subunit of IL-23 and inhibits its interaction with the IL-23 receptor. IL-23 is a naturally occurring cytokine that is involved in inflammatory and immune responses. Tildrakizumab inhibits the release of proinflammatory cytokines and chemokines.


BUROSUMAB

X-linked hypophosphatemia is caused by excess fibroblast growth factor 23 (FGF23) which suppresses renal tubular phosphate reabsorption and the renal production of 1,25 dihydroxy vitamin D. Burosumab-twza binds to and inhibits the biological activity of FGF23 restoring renal phosphate reabsorption and increasing the serum concentration of 1,25 dihydroxy vitamin D.


X-linked dominant hypophosphatemic rickets


Pronetupitant

Netupitant is a selective antagonist of human substance P/neurokinin 1 (NK-1) receptors. Delayed emesis has been largely associated with the activation of tachykinin family neurokinin 1 (NK-1) receptors (broadly distributed in the central and peripheral nervous systems) by substance P. As shown in in vitro and in vivo studies, netupitant inhibits substance P mediated responses.


Chemotherapy-induced nausea and vomiting


ERENUMAB

Erenumab-aooe is a human monoclonal antibody that binds to the calcitonin gene-related peptide (CGRP) receptor and antagonizes CGRP receptor function.


Avatrombopag

Avatrombopag is an orally bioavailable, small molecule TPO receptor agonist that stimulates proliferation and differentiation of megakaryocytes from bone marrow progenitor cells resulting in an increased production of platelets. Avatrombopag does not compete with TPO for binding to the TPO receptor and has an additive effect with TPO on platelet production.


Moxidectin

The mechanism by which moxidectin exhibits its effect against O. volvulus is not known. Studies with other nematodes suggest that moxidectin binds to glutamate-gated chloride channels (GluCl), gamma-aminobutyric acid (GABA) receptors and/or ATP-binding cassette (ABC) transporters. This leads to increased permeability, influx of chloride ions, hyperpolarization and muscle paralysis. Additionally, there is a reduction in motility of all stages of the parasite, excretion of immunomodulatory proteins, and the fertility of both male and female adult worms.


Plazomicin

Plazomicin is an aminoglycoside that acts by binding to bacterial 30S ribosomal subunit, thereby inhibiting protein synthesis. Plazomicin has concentration-dependent bactericidal activity as measured by time kill studies. In vitro studies demonstrated a plazomicin post-antibiotic effect ranging from 0.2 to 2.6 hours at 2X MIC against Enterobacteriaceae.


Cannabidiol

The precise mechanisms by which cannabidiol exerts its anticonvulsant effect in humans are unknown. Cannabidiol does not appear to exert its anticonvulsant effects through interaction with cannabinoid receptors.


Encorafenib

Encorafenib is a kinase inhibitor that targets BRAF V600E, as well as wild-type BRAF and CRAF. Mutations in the BRAF gene, such as BRAF V600E, can result in constitutively activated BRAF kinases that may stimulate tumor cell growth. Encorafenib was also able to bind to other kinases in vitro including JNK1, JNK2, JNK3, LIMK1, LIMK2, MEK4, and STK36 and substantially reduce ligand binding to these kinases at clinically achievable concentrations. Encorafenib inhibited in vitro growth of tumor cell lines expressing BRAF V600 E, D, and K mutations. In mice implanted with tumor cells expressing BRAF V600E, encorafenib induced tumor regressions associated with RAF/MEK/ERK pathway suppression.


Binimetinib

Binimetinib is a reversible inhibitor of mitogen-activated extracellular signal regulated kinase 1 (MEK1) and MEK2 activity. MEK proteins are upstream regulators of the extracellular signal-related kinase (ERK) pathway. In vitro, binimetinib inhibited extracellular signal-related kinase (ERK) phosphorylation in cellfree assays as well as viability and MEK-dependent phosphorylation of BRAF-mutant human melanoma cell lines. Binimetinib also inhibited in vivo ERK phosphorylation and tumor growth in BRAF-mutant murine xenograft models.


Dutetrabenazine

The precise mechanism by which deutetrabenazine exerts its anti-chorea effects is unknown but is believed to be related to its effect as a reversible depletor of monoamines (such as dopamine, serotonin, norepinephrine, and histamine) from nerve terminals. The major circulating metabolites (alpha-dihydrotetrabenazine [HTBZ] and beta-HTBZ) of deutetrabenazine, are reversible inhibitors of VMAT2, resulting in decreased uptake of monoamines into synaptic vesicles and depletion of monoamine stores.


Vaborbactam

Vaborbactam is a non-suicidal beta-lactamase inhibitor that protects meropenem from degradation by certain serine beta-lactamases such as Klebsiella pneumoniae carbapenemase (KPC). Vaborbactam does not have any antibacterial activity. Vaborbactam does not decrease the activity of meropenem against meropenem-susceptible organisms.


Urinary tract infection caused by Klebsiella


Urinary tract infection caused by Escherichia coli


COPANLISIB

Copanlisib is an inhibitor of phosphatidylinositol-3-kinase (PI3K) with inhibitory activity predominantly against PI3K-alpha and PI3K-delta isoforms expressed in malignant B cells. Copanlisib has been shown to induce tumor cell death by apoptosis and inhibition of proliferation of primary malignant B cell lines. Copanlisib inhibits several key cell-signaling pathways, including B-cell receptor (BCR) signaling, CXCR12 mediated chemotaxis of malignant B cells, and NFkB signaling in lymphoma cell lines.


Secnidazole

Secnidazole is a 5-nitroimidazole antimicrobial. 5-nitroimidazoles enter the bacterial cell as an inactive prodrug where the nitro group is reduced by bacterial enzymes to radical anions. It is believed that these radical anions interfere with bacterial DNA synthesis of susceptible isolates.


Butylphthalide

3-N-Butylphthalide (NBP) is a compound found in Chinese celery seed extracts that can improve cognitive functions and may decrease Amyloid-beta levels in Alzheimer′s disease. NBP has antioxidant activities and may protect against oxidative/nitrosative stress, mitochondrial impairment and apoptosis.


Abemaciclib

Abemaciclib is an inhibitor of cyclin-dependent kinases 4 and 6 (CDK4 and CDK6). These kinases are activated upon binding to D-cyclins. In estrogen receptor-positive (ER+) breast cancer cell lines, cyclin D1 and CDK4/6 promote phosphorylation of the retinoblastoma protein (Rb), cell cycle progression, and cell proliferation. In vitro, continuous exposure to abemaciclib inhibited Rb phosphorylation and blocked progression from G1 into S phase of the cell cycle, resulting in senescence and apoptosis. In breast cancer xenograft models, abemaciclib dosed daily without interruption as a single agent or in combination with antiestrogens resulted in reduction of tumor size.


Hormone receptor positive malignant neoplasm of breast


Acalabrutinib

Acalabrutinib is a small-molecule inhibitor of BTK. Acalabrutinib and its active metabolite, ACP-5862, form a covalent bond with a cysteine residue in the BTK active site, leading to inhibition of BTK enzymatic activity. BTK is a signaling molecule of the B cell antigen receptor (BCR) and cytokine receptor pathways. In B cells, BTK signaling results in activation of pathways necessary for B-cell proliferation, trafficking, chemotaxis, an  adhesion. In nonclinical studies, acalabrutinib inhibited BTK mediated activation of downstream signaling proteins CD86 and CD69 and inhibited malignant B-cell proliferation and survival.


Latanoprostene BUNOD

Latanoprostene bunod is thought to lower intraocular pressure by increasing outflow of aqueous humor through both the trabecular meshwork and uveoscleral routes. Intraocular pressure is a major modifiable risk factor for glaucoma progression. Reduction of intraocular pressure reduces risk of glaucomatous visual field loss.


Letermovir

Letermovir inhibits the CMV DNA terminase complex (pUL51, pUL56, and pUL89) which is required for viral DNA processing and packaging. Biochemical characterization and electron microscopy demonstrated that letermovir affects the production of proper unit length genomes and interferes with virion maturation. Genotypic characterization of virus resistant to letermovir confirmed that letermovir targets the terminase complex.


benralizumab

Benralizumab is a humanized afucosylated, monoclonal antibody (IgG1, kappa) that directly binds to the alpha subunit of the human interleukin-5 receptor (IL-5Ralpha) with a dissociation constant of 11 pM. The IL-5 receptor is expressed on the surface of eosinophils and basophils. In an in vitro setting, the absence of fucose in the Fc domain of benralizumab facilitates binding (45.5 nM) to FcɣRIII receptors on immune effectors cells, such as natural killer (NK) cells, leading to apoptosis of eosinophils and basophils through antibody-dependent cell-mediated cytotoxicity (ADCC). Inflammation is an important component in the pathogenesis of asthma. Multiple cell types (e.g., mast cells, eosinophils, neutrophils, macrophages, lymphocytes) and mediators (e.g., histamine, eicosanoids, leukotrienes, cytokines) are involved in inflammation. Benralizumab, by binding to the IL-5Ralpha chain, reduces eosinophils through ADCC; however, the mechanism of benralizumab action in asthma has not been definitively established.


vestronidase alfa

Mucopolysaccharidosis VII (MPS VII or Sly syndrome) is a lysosomal disorder characterized by the deficiency of GUS that results in GAG accumulation in cells throughout the body leading to multisystem tissue and organ damage. Vestronidase alfa-vjbk is a recombinant form of human GUS and is intended to provide exogenous GUS enzyme for uptake into cellular lysosomes. Mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow binding of the enzyme to cell surface receptors, leading to cellular uptake of the enzyme, targeting to lysosomes and subsequent catabolism of accumulated GAGs in affected tissues.


Rupatadine

Rupatadine is a second-generation antihistamine, long-acting histamine antagonist with selective peripheral H1-receptor and platelet activating factor (PAF) antagonistic activities. Some of the metabolites (desloratadine and its hydroxylated metabolites) retain an antihistaminic activity and may partially contribute to the overall efficacy of the drug, maintaining activity for up to 24 hours.


LONOCTOCOG ALFA

Lonoctocog alfa is a recombinant human protein that replaces the missing coagulation factor VIII needed for effective hemostasis. Lonoctocog alfa is a single polypeptide chain with a truncated B-domain that allows for a covalent bridge to link the factor VIII heavy and light chains. Lonoctocog alfa has demonstrated a higher VWF affinity relative to full-length rFVIII. VWF stabilizes factor VIII and protects it from degradation. Activated Lonoctocog alfa has an amino acid sequence identical to endogenous FVIIIa.


SEMAGLUTIDE

Semaglutide is a GLP-1 analogue with 94% sequence homology to human GLP-1. Semaglutide acts as a GLP-1 receptor agonist that selectively binds to and activates the GLP-1 receptor, the target for native GLP-1. GLP-1 is a physiological hormone that has multiple actions on glucose, mediated by the GLP-1 receptors. The principal mechanism of protraction resulting in the long half-life of semaglutide is albumin binding, which results in decreased renal clearance and protection from metabolic degradation. Furthermore, semaglutide is stabilized against degradation by the DPP-4 enzyme. Semaglutide reduces blood glucose through a mechanism where it stimulates insulin secretion and lowers glucagon secretion, both in a glucose-dependent manner. Thus, when blood glucose is high, insulin secretion is stimulated and glucagon secretion is inhibited. The mechanism of blood glucose lowering also involves a minor delay in gastric emptying in the early postprandial phase.


EMICIZUMAB

Emicizumab-kxwh is a humanized monoclonal modified immunoglobulin G4 (IgG4) antibody with a bispecific antibody structure binding factor IXa and factor X. Emicizumab-kxwh has an approximate molecular weight of 145.6 kDa and is produced in genetically engineered mammalian (Chinese hamster ovary) cells. Emicizumab-kxwh has no structural relationship or sequence homology to FVIII and, as such, does not induce or enhance the development of direct inhibitors to FVIII. Emicizumab-kxwh bridges activated factor IX and factor X to restore the function of missing activated factor VIII that is needed for effective hemostasis.


Ozenoxacin

Ozenoxacin is a quinolone antimicrobial drug. The mechanism of action involves the inhibition of bacterial DNA replication enzymes, DNA gyrase A and topoisomerase IV. Ozenoxacin has been shown to be bactericidal against S. aureus and S. pyogenes organisms. Ozenoxacin is used as topical treatment for impetigo and other infectious dermatological conditions.


Amenamevir

Amenamevir is an anti-herpes virus agent for the treatment of herpes zoster (shingles) with a novel mechanism of action. Amenamevir has been observed to inhibit the proliferation of the varicella-zoster virus by inhibiting the activity of the helicase-primase complex, which is essential for viral DNA replication.


Netarsudil

Netarsudil is a rho kinase inhibitor, which is believed to reduce IOP by increasing the outflow of aqueous humor through the trabecular meshwork route.


Tivozanib

Tivozanib is an oral, once-daily, vascular endothelial growth factor (VEGF) tyrosine kinase inhibitor (TKI). It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications. Tivozanib is indicated as first line treatment of adult patients with advanced renal cell carcinoma (RCC) and for adult patients who are VEGFR and mTOR pathway inhibitor-naive following disease progression after one prior treatment with cytokine therapy for advanced RCC.


Forodesine

Forodesine is a transition state analogue that binds preferentially to and inhibits purine nucleotide phosphorylase (PNP), resulting in the accumulation of deoxyguanosine triphosphate and the subsequent inhibition of the enzyme ribonucleoside diphosphate reductase and DNA synthesis. This agent selectively causes apoptosis in stimulated or malignant T-lymphocytes. A transition state analogue is a substrate designed to mimic the properties or the geometry of the transition state of reaction.


mature T-cell and NK-cell non-Hodgkin lymphoma


Regorafenib

Regorafenib is a small molecule inhibitor of multiple membrane-bound and intracellular kinases involved in normal cellular functions and in pathologic processes such as oncogenesis, tumor angiogenesis, metastasis and tumor immunity. In in vitro biochemical or cellular assays, regorafenib or its major human active metabolites M-2 and M-5 inhibited the activity of RET, VEGFR1, VEGFR2, VEGFR3, KIT, PDGFR-alpha, PDGFR-beta, FGFR1, FGFR2, TIE2, DDR2, TrkA, Eph2A, RAF-1, BRAF, BRAF V600E, SAPK2, PTK5, Abl and CSF1R at concentrations of regorafenib that have been achieved clinically. In in vivo models, regorafenib demonstrated anti-angiogenic activity in a rat tumor model and inhibition of tumor growth in several mouse xenograft models including some for human colorectal carcinoma, gastrointestinal stromal and hepatocellular carcinoma. Regorafenib also demonstrated anti-metastatic activity in a mouse xenograft model and two mouse orthotopic models of human colorectal carcinoma.


Midostaurin

Midostaurin is a small molecule that inhibits multiple receptor tyrosine kinases. In vitro biochemical or cellular assays have shown that midostaurin or its major human active metabolites CGP62221 and CGP52421 inhibit the activity of wild type FLT3, FLT3 mutant kinases (ITD and TKD), KIT (wild type and D816V mutant), PDGFR-alfa/beta, VEGFR2, as well as members of the serine/threonine kinase PKC (protein kinase C) family. Midostaurin demonstrated the ability to inhibit FLT3 receptor signaling and cell proliferation, and it induced apoptosis in leukemic cells expressing ITD and TKD mutant FLT3 receptors or overexpressing wild type FLT3 and PDGF receptors. Midostaurin also demonstrated the ability to inhibit KIT signaling, cell proliferation and histamine release and induce apoptosis in mast cells.


cerliponase alfa

Cerliponase alfa is a purified human enzyme produced by recombinant DNA technology in a Chinese hamster ovary cell line. The active substance is a recombinant human tripeptidyl peptidase-1 (rhTPP1), a lysosomal exopeptidase. The primary activity of the mature enzyme is the cleavage of N-terminal tripeptides from a broad range of protein substrates. CLN2 disease is a neurodegenerative disease caused by deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1), which catabolizes polypeptides in the CNS. Deficiency in TPP1 activity results in the accumulation of lysosomal storage materials normally metabolized by this enzyme in the central nervous system (CNS), leading to progressive decline in motor function.


late infantile neuronal ceroid lipofuscinosis


ABALOPARATIDE

Abaloparatide is a PTHrP(1-34) analog which acts as an agonist at the PTH1 receptor (PTH1R). This results in activation of the cAMP signaling pathway in target cells. In rats and monkeys, abaloparatide had an anabolic effect on bone, demonstrated by increases in BMD and bone mineral content (BMC) that correlated with increases in bone strength at vertebral and/or nonvertebral sites.


Brigatinib

Brigatinib is a tyrosine kinase inhibitor with in vitro activity at clinically achievable concentrations against multiple kinases including ALK, ROS1, insulin-like growth factor-1 receptor (IGF-1R), and FLT-3 as well as EGFR deletion and point mutations. Brigatinib inhibited autophosphorylation of ALK and ALK-mediated phosphorylation of the downstream signaling proteins STAT3, AKT, ERK1/2, and S6 in in vitro and in vivo assays. Brigatinib also inhibited the in vitro proliferation of cell lines expressing EML4-ALK and NPM-ALK fusion proteins and demonstrated dose-dependent inhibition of EML4-ALK-positive NSCLC xenograft growth in mice. At clinically achievable concentrations (<= 500 nM), brigatinib inhibited the in vitro viability of cells expressing EML4-ALK and 17 mutant forms associated with resistance to ALK inhibitors including crizotinib, as well as EGFR-Del (E746-A750), ROS1-L2026M, FLT3-F691L, and FLT3-D835Y. Brigatinib exhibited in vivo anti-tumor activity against 4 mutant forms of EML4-ALK, including G1202R and L1196M mutants identified in NSCLC tumors in patients who have progressed on crizotinib. Brigatinib also reduced tumor burden and prolonged survival in mice implanted intracranially with an ALK-driven tumor cell line.


DURVALUMAB

Durvalumab is a human immunoglobulin G1 kappa (IgG1κ) monoclonal antibody that blocks the interaction of PD-L1 with PD-1 and CD80 (B7.1). Blockade of PD-L1/PD-1 and PD-L1/CD80 interactions releases the inhibition of immune responses, without inducing antibody dependent cellmediated cytotoxicity (ADCC). PD-L1 blockade with durvalumab led to increased T-cell activation in vitro and decreased tumor size in co-engrafted human tumor and immune cell xenograft mouse models.


a humanised anti-interleukin-6 receptor monoclonal antibody


cenegermin

Cenegermin is a recombinant form of human nerve growth factor. Nerve growth factor is an endogenous protein involved in the differentiation and maintenance of neurons, which acts through specific high-affinity (i.e., TrkA) and low-affinity (i.e. p75NTR) nerve growth factor receptors. Nerve growth factor receptors are expressed in the anterior segment of the eye (cornea, conjunctiva, iris, ciliary body, and lens), by the lacrimal gland, and by posterior segment intraocular tissues. The treatment with cenegermin, administered as eye drops, is intended to allow restoration of corneal integrity.


sarilumab

Sarilumab is a human recombinant monoclonal antibody of the IgG1 subclass. Sarilumab is produced by recombinant DNA technology in Chinese Hamster Ovary cell suspension culture. Sarilumab binds to both soluble and membrane-bound IL-6 receptors (sIL-6R and mIL-6R), and has been shown to inhibit IL-6-mediated signaling through these receptors. IL-6 is a pleiotropic pro-inflammatory cytokine produced by a variety of cell types including T- and B-cells, lymphocytes, monocytes, and fibroblasts. IL-6 has been shown to be involved in diverse physiological processes such as T-cell activation, induction of immunoglobulin secretion, initiation of hepatic acute phase protein synthesis, and stimulation of hematopoietic precursor cell proliferation and differentiation. IL-6 is also produced by synovial and endothelial cells leading to local production of IL-6 in joints affected by inflammatory processes such as rheumatoid arthritis. Based on encouraging results in China with a similar drug, tocilizumab, a phase 2/3 US study evaluating efficacy and safety of sarilumab in patients hospitalized with severe COVID-19 was performed. At the moment, there are insufficient data to recommend either for or against use of sarilumab for hospitalized patients with COVID-19.


Sitafloxacin

Sitafloxacin hydrate is a fluoroquinolone antibiotic. It is indicated for the treatment of various infections such as respiratory infection, urologic infection, gynecologic infection, otorhinological infections, and dental infection.


Delafloxacin

Delafloxacin belongs to the fluoroquinolone class of antibacterial drugs and is anionic in nature. The antibacterial activity of delafloxacin is due to the inhibition of both bacterial topoisomerase IV and DNA gyrase (topoisomerase II) enzymes which are required for bacterial DNA replication, transcription, repair, and recombination. Delafloxacin exhibits a concentration-dependent bactericidal activity against gram-positive and gram-negative bacteria in vitro.


Polmacoxib

Polmacoxib is a nonsteroidal anti-inflammatory drug (NSAID) used to treat osteoarthritis. It is a selective COX-2 inhibitor.


Evogliptin

Evogliptin is an orally bioavailable, selective dipeptidyl peptidase-4 inhibitor for the treatment of type 2 diabetes mellitus. DPP-4 inhibitors control glucose levels by preventing the breakdown of the incretin hormones glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide-1 (GLP-1), which stimulate insulin secretion in response to the increased levels of glucose in the period following meals.


Benproperine

has antineoplastic activity; structure in first source


Betrixaban

Betrixaban is an oral FXa inhibitor that selectively blocks the active site of FXa and does not require a cofactor (such as Anti-thrombin III) for activity. Betrixaban inhibits free FXa and prothrombinase activity. By directly inhibiting FXa, betrixaban decreases thrombin generation. Betrixaban has no direct effect on platelet aggregation.


Cladribine

An antineoplastic agent used in the treatment of lymphoproliferative diseases including hairy-cell leukemia.


Voxilaprevir

Voxilaprevir is a noncovalent, reversible inhibitor of the NS3/4A protease, which is necessary for the proteolytic cleavage of the HCV encoded polyprotein (into mature forms of the NS3, NS4A, NS4B, NS5A, and NS5B proteins) and is essential for viral replication. In a biochemical inhibition assay, voxilaprevir inhibited the proteolytic activity of recombinant NS3/4A enzymes from clinical isolates of HCV genotypes 1b and 3a with Ki values of 38 and 66 pM, respectively.


Glecaprevir

Glecaprevir is an inhibitor of the HCV NS3/4A protease, which is necessary for the proteolytic cleavage of the HCV encoded polyprotein (into mature forms of the NS3, NS4A, NS4B, NS5A, and NS5B proteins) and is essential for viral replication. In a biochemical assay, glecaprevir inhibited the proteolytic activity of recombinant NS3/4A enzymes from clinical isolates of HCV genotypes 1a, 1b, 2a, 2b, 3a, 4a, 5a, and 6a with IC50 values ranging from 3.5 to 11.3 nM.


Pibrentasvir

Pibrentasvir is an inhibitor of HCV NS5A, which is essential for viral RNA replication and virion assembly. The mechanism of action of pibrentasvir has been characterized based on cell culture antiviral activity and drug resistance mapping studies.


Rucaparib

Rucaparib is an inhibitor of poly (ADP-ribose) polymerase (PARP) enzymes, including PARP-1, PARP-2, and PARP-3, which play a role in DNA repair. In vitro studies have shown that rucaparib-induced cytotoxicity may involve inhibition of PARP enzymatic activity and increased formation of PARP-DNA complexes resulting in DNA damage, apoptosis, and cell death. Increased rucaparib-induced cytotoxicity was observed in tumor cell lines with deficiencies in BRCA1/2 and other DNA repair genes. Rucaparib has been shown to decrease tumor growth in mouse xenograft models of human cancer with or without deficiencies in BRCA.


Baricitinib

Baricitinib is a selective and reversible inhibitor of Janus kinase (JAK)1 and JAK2. Janus kinases (JAKs) are enzymes that transduce intracellular signals from cell surface receptors for a number of cytokines and growth factors involved in haematopoiesis, inflammation and immune function. Within the intracellular signalling pathway, JAKs phosphorylate and activate signal transducers and activators of transcription (STATs), which activate gene expression within the cell. Baricitinib modulates these signalling pathways by partially inhibiting JAK1 and JAK2 enzymatic activity, thereby reducing the phosphorylation and activation of STATs. FDA issued an Emergency use authorization (EUA) on November 19, 2020 that permits use of baricitinib in combination with remdesivir for treatment of suspected or laboratory-confirmed COVID-19 in hospitalized patients ≥2 years of age requiring supplemental oxygen, invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO).


Acetyldigitoxin

Cardioactive derivatives of lanatoside A or of DIGITOXIN. They are used for fast digitalization in congestive heart failure.


6-Acetamidohexanoic acid

wound-healing agent component of plasmutan


Crisaborole

Crisaborole is a phosphodiesterase 4 (PDE-4) inhibitor. PDE-4 inhibition results in increased intracellular cyclic adenosine monophosphate (cAMP) levels. The specific mechanism(s) by which crisaborole exerts its therapeutic action for the treatment of atopic dermatitis is not well defined.


ferumoxytol

Ferumoxytol consists of a superparamagnetic iron oxide that is coated with a carbohydrate shell, which helps to isolate the bioactive iron from plasma components until the iron-carbohydrate complex enters the reticuloendothelial system macrophages of the liver, spleen and bone marrow. The iron is released from the iron-carbohydrate complex within vesicles in the macrophages. Iron then either enters the intracellular storage iron pool (e.g., ferritin) or is transferred to plasma transferrin for transport to erythroid precursor cells for incorporation into hemoglobin.


PEGADEMASE BOVINE

Pegademase bovine is a modified enzyme used for enzyme replacement therapy for the treatment of severe combined immunodeficiency disease (SCID) associated with a deficiency of adenosine deaminase.  In the absence of the ADA enzyme, the purine substrates adenosine and 2'-deoxyadenosine accumulate, causing metabolic abnormalities that are directly toxic to lymphocytes.


severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency


Interferon alfa-2B

Interferon alfa-2b is an alpha interferon and is a water-soluble protein with a molecular weight of 19,271 daltons produced by recombinant DNA techniques. Interferons exert their cellular activities by binding to specific membrane receptors on the cell surface. Once bound to the cell membrane, interferons initiate a complex sequence of intracellular events. In vitro studies demonstrated that these include the induction of certain enzymes, suppression of cell proliferation, immunomodulating activities such as enhancement of the phagocytic activity of macrophages and augmentation of the specific cytotoxicity of lymphocytes for target cells, and inhibition of virus replication in virus-infected cells. Interferon alfa via inhalation is included in national guidelines from China as a possible option for treatment of COVID-19.


anogenital human papillomavirus infection


Nusinersen

Nusinersen is a modified antisense oligonucleotide, where the 2' hydroxy groups of the ribofuranosyl rings are replaced with 2'-O-2-methoxyethyl groups and the phosphate linkages are replaced with phosphorothioate linkages. Nusinersen binds to a specific sequence in the intron downstream of exon 7 of the SMN2 transcript. Nusinersen is designed to treat spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, SPINRAZA was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein.


Oxymetazoline

Imidazoline derivative with sympathomimetic activity that stimulates alpha-adrenergic receptors in the arterioles of the nasal mucosa to produce vasoconstriction.


Imolamine

blood platelet aggregation antagonist; RN given refers to parent cpd; structure


Plecanatide

Plecanatide is structurally related to human uroguanylin, and similar to uroguanylin, plecanatide functions as a guanylate cyclase-C (GC-C) agonist. Both plecanatide and its active metabolite bind to GC-C and act locally on the luminal surface of the intestinal epithelium. Activation of GC-C results in an increase in both intracellular and extracellular concentrations of cyclic guanosine monophosphate (cGMP). Elevation of intracellular cGMP stimulates secretion of chloride and bicarbonate into the intestinal lumen, mainly through activation of the cystic fibrosis transmembrane conductance regulator (CFTR) ion channel, resulting in increased intestinal fluid and accelerated transit.


Olmutinib

Olmutinib is an oral, third-generation epidermal growth factor receptor tyrosine kinase inhibitor (EGFR TKI) for the treatment of non-small cell lung cancer (NSCLC). Third-generation EGFR TKIs with covalent binding to the receptors demonstrate irreversible enzymatic inhibition of activating EGFR mutations and T790M mutation (a common reason for acquired EGFR TKI resistance), while sparing wild-type EGFR.


Icotinib

Icotinib is a new epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) that developed and used in China for the treatment of patients with non-small cell lung cancer (NSCLC).


Radotinib

A second-generation tyrosine kinase inhibitor of Bcr-Abl fusion protein and the platelet-derived growth factor receptor (PDGFR), with potential antineoplastic activity. Upon administration, radotinib specifically inhibits the Bcr-Abl fusion protein, an abnormal enzyme expressed in Philadelphia chromosome-positive chronic myeloid leukemia (CML) cells. In addition, this agent also inhibits PDGFR thereby blocking PDGFR-mediated signal transduction pathways. The inhibitory effect of radotinib on these specific tyrosine kinases may decrease cellular proliferation and inhibit angiogenesis. This agent has shown potent efficacy in CML cells that are resistant to the first-generation standard tyrosine kinase inhibitors, such as imatinib, nilotinib and dasatinib.


chronic myelogenous leukemia, BCR-ABL1 positive


Dehydroepiandrosterone

A major C19 steroid produced by the ADRENAL CORTEX. It is also produced in small quantities in the TESTIS and the OVARY. Dehydroepiandrosterone (DHEA) can be converted to TESTOSTERONE; ANDROSTENEDIONE; ESTRADIOL; and ESTRONE. Most of DHEA is sulfated (DEHYDROEPIANDROSTERONE SULFATE) before secretion.


ALBUTREPENONACOG ALFA

derived from recombinant factor IX and recombinant human albumin; used to treat hemophilia B


Lenvatinib

a receptor tyrosine kinase (RTK) inhibitor that inhibits the kinase activities of vascular endothelial growth factor (VEGF) receptors VEGFR1 (FLT1), VEGFR2 (KDR), and VEGFR3 (FLT4)


Ciprofibrate

Ciprofibrate is a new derivative of phenoxyisobutyric acid which has a marked hypolipidaemic action. It reduces both LDL and VLDL and hence the levels of triglyceride and cholesterol associated with these lipoprotein fractions. It also increases levels of HDL cholesterol.


hyperlipidemia, familial combined, LPL related


TALIGLUCERASE ALFA

Taliglucerase alfa, a long term enzyme replacement therapy, is a recombinant analog of human lysosomal glucocerebrosidase that catalyzes the hydrolysis of glucocerebroside to glucose and ceramide, reducing the amount of accumulated glucocerebroside. Taliglucerase alfa uptake into cellular lysosomes is mediated by binding of Taliglucerase alfa mannose oligosaccharide chains to specific mannose receptors on the cell surface leading to internalization and subsequent transport to the lysosomes.


Pimavanserin

an atypical antipsychotic, mechanism of action of pimavanserin in the treatment of hallucinations and delusions associated with Parkinson's disease psychosis is unknown. however, the effect of pimavanserin could be mediated through a combination of inverse agonist and antagonist activity at serotonin 5-HT2A receptors and to a lesser extent at serotonin 5-HT2C receptors


Histamine

An amine derived by enzymatic decarboxylation of HISTIDINE. It is a powerful stimulant of gastric secretion, a constrictor of bronchial smooth muscle, a vasodilator, and also a centrally acting neurotransmitter.


Empagliflozin

Empagliflozin is an inhibitor of SGLT2. By inhibiting SGLT2, empagliflozin reduces renal reabsorption of filtered glucose and lowers the renal threshold for glucose, and thereby increases urinary glucose excretion.


Aceclidine

cholinomimetic used to reduce intraocular pressure in glaucoma


Acefylline

Acefylline is a bronchodilator agent, relaxing brochial smooth muscle and opposing the effects of many broncho-constrictor mediators. Also, it relaxes smooth muscle in the urinary and biliary ways.


Acenocoumarol

A coumarin that is used as an anticoagulant. Its actions and uses are similar to those of WARFARIN. (From Martindale, The Extra Pharmacopoeia, 30th ed, p233)


Acetyl-L-carnitine

An acetic acid ester of CARNITINE that facilitates movement of ACETYL COA into the matrices of mammalian MITOCHONDRIA during the oxidation of FATTY ACIDS.


bezlotoxumab

Bezlotoxumab is a human monoclonal antibody that binds C. difficile toxin B. Bezlotoxumab inhibits the binding of toxin B and prevents its effects on mammalian cells. Bezlotoxumab does not bind to C. difficile toxin A.


olaratumab

Olaratumab is a human IgG1 antibody that binds platelet-derived growth factor receptor alpha (PDGFR-alpha). PDGFR-alpha is a receptor tyrosine kinase expressed on cells of mesenchymal origin. Signaling through this receptor plays a role in cell growth, chemotaxis, and mesenchymal stem cell differentiation. The receptor has also been detected on some tumor and stromal cells, including sarcomas, where signaling can contribute to cancer cell proliferation, metastasis, and maintenance of the tumor microenvironment. The interaction between olaratumab and PDGFR-alpha prevents binding of the receptor by the PDGF-AA and -BB ligands as well as PDGF-AA, -BB, and -CC-induced receptor activation and downstream PDGFR-alpha pathway signaling. Olaratumab exhibits in vitro and in vivo anti-tumor activity against selected sarcoma cell lines and disrupted the PDGFR-alpha signaling pathway in in vivo tumor implant models.


Chloroxylenol

a antiseptic halogenated phenol, non-toxic, non-corrosive, non-staining with high phenol coefficient, it may be applied directly to a wound and shows no chemical reactivity toward blood


sympathomimetic aliphatic amine with vasoconstrictor activity


Motretinide

exerts a therapeutic influence on chemically induced papillomas & carcinomas of the skin in mice; RN given refers to cpd without isomeric designation; structure


METHACYCLINE

A broad-spectrum semisynthetic antibiotic related to TETRACYCLINE but excreted more slowly and maintaining effective blood levels for a more extended period.


Lifitegrast

a lymphocyte function-associated antigen-1 ( LFA-1) antagonist, blocks the interaction of LFA-1 with its cognate ligand intercellular adhesion molecule-1 (ICAM-1), LFA-1/ICAM-1 interaction can contribute to the formation of an immunological synapse resulting in T-cell activation and migration to target tissues


EPTOTERMIN ALFA

an osteoinductive initiates bone formation through the induction of cellular differentiation in mesenchymal cells, which are recruited to the implant site from bone marrow periosteum and muscle, used for treatment of non-union of bone fractures


Oxyphencyclimine

cholinergic blocking agent used as an adjunct in the treatment of peptic ulcer


Lisuride

An ergot derivative that acts as an agonist at dopamine D2 receptors (DOPAMINE AGONISTS). It may also act as an antagonist at dopamine D1 receptors, and as an agonist at some serotonin receptors (SEROTONIN RECEPTOR AGONISTS).


Danthron

mild purgative anthraquinone derivative chemically related to emodin, the active principle of cascara and other naturally occurring products such as senna, aloes and rhubarb, it acts on the nerve endings of the myenteric plexus and stimulates the muscles of the large intestine


PEGLOTICASE

a recombinant uricase and achieves its therapeutic effect by catalyzing the oxidation of uric acid to allantoin, thereby lowering serum uric acid


Clidinium

a synthetic anticholinergic agent that has antispasmodic and antisecretory effect on the gastrointestinal tract


Encainide

One of the ANTI-ARRHYTHMIA AGENTS, it blocks VOLTAGE-GATED SODIUM CHANNELS and slows conduction within the His-Purkinje system and MYOCARDIUM.


Posaconazole

an antifungal medicine that belongs to the triazole group, it works by preventing the formation of ergosterol, which is an important part of fungal cell walls


Oxyphenisatin acetate

A laxative that undergoes enterohepatic circulation. It may cause jaundice.


PEGINTERFERON BETA-1A

an interferon beta-1a to which a single, linear 20,000 dalton methoxy poly(ethyleneglycol)-O-2-methylpropionaldehyde molecule is covalently attached to the alpha amino group of the N-terminal amino acid residue. A possible role for IFNs in prophylaxis or early treatment of COVID-19 has been suggested to compensate for possibly insufficient endogenous IFN production. However, the efficacy and safety of IFNs for treatment or prevention of COVID-19 were not established.


INTERFERON BETA-1A

An interferon beta-1 subtype that has a methionine at position 1, a cysteine at position 17, and is glycosylated at position 80. It functions as an ANTI-VIRAL AGENT and IMMUNOMODULATOR and is used to manage the symptoms of RELAPSING-REMITTING MULTIPLE SCLEROSIS. A possible role for IFNs in prophylaxis or early treatment of COVID-19 has been suggested to compensate for possibly insufficient endogenous IFN production. However, the efficacy and safety of IFNs for treatment or prevention of COVID-19 were not established.


INTERFERON BETA-1B

A non-glycosylated form of interferon beta-1 that has a serine at position 17. It is used in the treatment of both RELAPSING-REMITTING MULTIPLE SCLEROSIS and CHRONIC PROGRESSIVE MULTIPLE SCLEROSIS. A possible role for IFNs in prophylaxis or early treatment of COVID-19 has been suggested to compensate for possibly insufficient endogenous IFN production. However, the efficacy and safety of IFNs for treatment or prevention of COVID-19 were not established.


Edaravone

Edaravone protects neurons from oxidative stress by scavenging free radicals that may cause cellular damage, and this mechanism is expected to be effective both in patients with acute cerebral infarction and in those with ALS


alteplase

A proteolytic enzyme in the serine protease family found in many tissues which converts PLASMINOGEN to FIBRINOLYSIN. It has fibrin-binding activity and is immunologically different from UROKINASE-TYPE PLASMINOGEN ACTIVATOR. The primary sequence, composed of 527 amino acids, is identical in both the naturally occurring and synthetic proteases. Is currently clinical tested as  treatment for COVID-19 patients.


Cabozantinib

Cabozantinib inhibits the tyrosine kinase activity of MET, VEGFR-1, -2 and -3, AXL, RET, ROS1, TYRO3, MER, KIT, TRKB, FLT-3, and TIE-2. These receptor tyrosine kinases are involved in both normal cellular function and pathologic processes such as oncogenesis, metastasis, tumor angiogenesis, drug resistance, and maintenance of the tumor microenvironment.


Opicapone

a peripheral, selective and reversible catechol-O-methyltransferase (COMT) inhibitor endowed with a high binding affinity (sub-picomolar) that translates into a slow complex dissociation rate constant and a long duration of action (>24 hours) in vivo


Faropenem daloxate

a faropenem prodrug for treating bacterial infections


FERUMOXSIL

a large superparamagnetic iron oxide colloid; a miscible darkening agent; see also the dextran-coated superparamagnetic iron oxides - ferumoxides and ferumoxtran


PORACTANT ALFA

Endogenous pulmonary surfactant (isolated from porcine lungs) reduces surface tension at the air-liquid interface of the alveoli during ventilation and stabilizes the alveoli against collapse at resting transpulmonary pressures. A deficiency of pulmonary surfactant in preterm infants results in Respiratory Distress Syndrome (RDS) characterized by poor lung expansion, inadequate gas exchange, and a gradual collapse of the lungs (atelectasis).


Talipexole

dopamine receptor agonist; structure given in first source


Venetoclax

Venetoclax is a selective and orally bioavailable small-molecule inhibitor of BCL-2, an anti-apoptotic protein. Overexpression of BCL-2 has been demonstrated in CLL cells where it mediates tumor cell survival and has been associated with resistance to chemotherapeutics. Venetoclax helps restore the process of apoptosis by binding directly to the BCL-2 protein, displacing pro-apoptotic proteins like BIM, triggering mitochondrial outer membrane permeabilization and the activation of caspases. In nonclinical studies, venetoclax has demonstrated cytotoxic activity in tumor cells that overexpress BCL-2.


dornase alfa

A recombinant human deoxyribonuclease I (rhDNAse) with selective DNA cleaving activity. Upon intrapleural administration, dornase alfa catalyzes the degradation of extracellular DNA in airway secretions, which can reduce their viscosity. Thus, dornase alfa may both promote the clearing of airway mucus and improve pulmonary function.


Lofexidine

Lofexidine is a central alpha-2 adrenergic agonist that binds to receptors on adrenergic neurons. This reduces the release of norepinephrine and decreases sympathetic tone.


Eribulin

Eribulin inhibits the growth phase of microtubules without affecting the shortening phase and sequesters tubulin into nonproductive aggregates. Eribulin exerts its effects via a tubulin-based antimitotic mechanism leading to G2/M cell-cycle block, disruption of mitotic spindles, and, ultimately, apoptotic cell death after prolonged mitotic blockage. In addition, eribulin treatment of human breast cancer cells caused changes in morphology and gene expression as well as decreased migration and invasiveness in vitro. In mouse xenograft models of human breast cancer, eribulin treatment was associated with increased vascular perfusion and permeability in the tumor cores, resulting in reduced tumor hypoxia, and changes in the expression of genes in tumor specimens associated with a change in phenotype.


Pegaspargase

covalent attachment of PEG to enzyme produces active, stable complex; non-immunogenic; used in treatment of leukemia


Elbasvir

inhibits NS5A protein of hepatitis C virus


Oxetorona

treatment of migraine; RN given refers to parent cpd; structure


Artemisinin

antimalarial from Chinese medicinal herb Artemisia annua


IXEKIZUMAB

A humanized anti-interleukin 17 monoclonal antibody used for the treatment of PSORIASIS.


eftrenonacog alfa

consists of factor IX fused to the Fc fragment of human IgG1


besilesomab

a mouse monoclonal antibody labelled with the radioactive isotope technetium-99m for the detection of septic arthroplasty


Racecadotril

A potent inhibitor of membrane metalloendopeptidase (ENKEPHALINASE). Thiorphan potentiates morphine-induced ANALGESIA and attenuates naloxone-precipitated withdrawal symptoms.


DENILEUKIN DIFTITOX

more potent, truncated form of DAB(486)-IL-2; inhibit HIV-1 RNA production in infected cells


primary cutaneous T-cell non-Hodgkin lymphoma


alefacept

A recombinant human fusion protein of LFA-3 antigen and immunoglobulin IGG1 that binds to CD2 ANTIGENS on memory T-LYMPHOCYTES, preventing their activation and proliferation. It is used in the management of moderate to severe chronic plaque PSORIASIS.


ABATACEPT

A fusion protein immunoconjugate of the extracellular domain of CTLA4 and the Fc domain of human IgG1. It functions as a T-cell co-stimulation blocker that inhibits TNF-ALPHA and prevents the activation of T-LYMPHOCYTES. It is used in the treatment of RHEUMATOID ARTHRITIS.


Nafamostat

inhibitor of trypsin, plasmin, pancreatic kallikrein, plasma kallikrein & thrombin; strongly inhibits esterolytic activities of C1r & C1 esterase complement-mediated hemolysis; antineoplastic; RN given refers to parent cpd


Climbazole

Climbazole is a conazole fungicide. It is also use in shampoo and cleaner as an anti-dandruff agents and anti-bacteria agent.


Tenecteplase

A tissue plasminogen activator enzyme that acts as a fibrinolytic agent; it is used for the dissolution of blood clots, such as those that occur in acute MYOCARDIAL INFARCTION.


Ixazomib

an oral proteasome inhibitor with antineoplastic activity; MLN2238 is the biologically active form of MLN9708


Brivaracetam

Brivaracetam displays a high and selective affinity for synaptic vesicle protein 2A (SV2A) in the brain, which may contribute to the anticonvulsant effect.


DARATUMUMAB

Daratumumab is an IgG1k human monoclonal antibody (mAb) that binds to CD38 and inhibits the growth of CD38 expressing tumor cells by inducing apoptosis directly through Fc mediated cross linking as well as by immune-mediated tumor cell lysis through complement dependent cytotoxicity (CDC), antibody dependent cell mediated cytotoxicity (ADCC) and antibody dependent cellular phagocytosis (ADCP).


susoctocog alfa

a recombinant B-domain deleted porcine sequence Factor VIII, binds to von Willebrand factor


Asparaginase

A hydrolase enzyme that converts L-asparagine and water to L-aspartate and NH3. EC 3.5.1.1.


nivolumab

A genetically engineered, fully humanized immunoglobulin G4 monoclonal antibody that binds to the PD-1 RECEPTOR, activating an immune response to tumor cells. It is used as monotherapy or in combination with IPILIMUMAB for the treatment of advanced malignant MELANOMA. It is also used in the treatment of advanced or recurring NON-SMALL CELL LUNG CANCER; RENAL CELL CARCINOMA; and HODGKIN'S LYMPHOMA.


necitumumab

a fully human antibody that has similar antitumor potency as the chimeric cetuximab/Erbitux and might represent a safer therapeutic alternative in EGF receptor inhibition


adalimumab

A humanized monoclonal antibody that binds specifically to TNF-ALPHA and blocks its interaction with endogenous TNF RECEPTORS to modulate INFLAMMATION. It is used in the treatment of RHEUMATOID ARTHRITIS; PSORIATIC ARTHRITIS; CROHN'S DISEASE and ULCERATIVE COLITIS.


ELOTUZUMAB

a humanized monoclonal antibody that selectively targets glycoproteins expressed on myeloma cells


Felbinac

an anti-inflammatory/analgesic agent which has been developed into a topical gel for local treatment and pain and inflammation associated with conditions of the musculo-skeletal system


Echothiophate

A potent, long-acting cholinesterase inhibitor used as a miotic in the treatment of glaucoma.


Lorcaserin

a serotonin 2C receptor agonist for oral administration used for chronic weight management


Uridine triacetate is an acetylated form of uridine. Following oral administration, uridine triacetate is deacetylated by nonspecific esterases present throughout the body, yielding uridine in the systemic circulation of patients with hereditary orotic aciduria who cannot synthesize adequate quantities of uridine due to a genetic defect in uridine nucleotide synthesis.


Lesinurad

Lesinurad reduces serum uric acid levels by inhibiting the function of transporter proteins involved in uric acid reabsorption in the kidney. Lesinurad inhibited the function of two apical transporters responsible for uric acid reabsorption, uric acid transporter 1 (URAT1) and organic anion transporter 4 (OAT4), with IC50 values of 7.3 and 3.7 μM, respectively. URAT1 is responsible for the majority of the reabsorption of filtered uric acid from the renal tubular lumen. OAT4 is a uric acid transporter associated with diuretic-induced hyperuricemia.


Alclofenac

Alclofenac is a non-steroidal anti-inflammatory agent, withdrawn from the market. It acts by blocking the synthesis of prostaglandins through the inhibition of cyclooxygenase, which converts arachidonic acid to cyclic endoperoxides, precursors of prostaglandins. Inhibition of prostaglandin synthesis accounts for their analgesic, antipyretic, and platelet-inhibitory actions.


Cobimetinib

Cobimetinib is a reversible inhibitor of mitogen-activated protein kinase (MAPK)/extracellular signal regulated kinase 1 (MEK1) and MEK2. MEK proteins are upstream regulators of the extracellular signal-related kinase (ERK) pathway, which promotes cellular proliferation. BRAF V600E and K mutations result in constitutive activation of the BRAF pathway which includes MEK1 and MEK2. In mice implanted with tumor cell lines expressing BRAF V600E, cobimetinib inhibited tumor cell growth.


Aprindine

A class Ib anti-arrhythmia agent used to manage ventricular and supraventricular arrhythmias.


Dyslipidemia due to type 2 diabetes mellitus


Pitolisant

Pitolisant is a potent, orally active histamine H3-receptor antagonist/inverse agonist which, via its blockade of histamine auto-receptors enhances the activity of brain histaminergic neurons, a major arousal system with widespread projections to the whole brain. Pitolisant also modulates various neurotransmitter systems, increasing acetylcholine, noradrenaline and dopamine release in the brain.


asfotase alfa

An enzyme that catalyzes the conversion of an orthophosphoric monoester and water to an alcohol and orthophosphate. EC 3.1.3.1.


astodrimer

microbicide, polyanion-based entry inhibitor, astodrimer is a broad-spectrum dendrimer consisting of a divalent benzhydrylamine core and four layers of lysine branches


COLLAGENASE CLOSTRIDIUM HISTOLYTICUM

purified collagenase clostridium histolyticum, consisting of two microbial collagenases in a defined mass ratio, Collagenase AUX-I and Collagenase AUX-II, which are isolated and purified from the fermentation of Clostridium histolyticum bacteria


Dupuytren's disease of finger, with contracture


Sufentanil

An opioid analgesic that is used as an adjunct in anesthesia, in balanced anesthesia, and as a primary anesthetic agent.


PEMBROLIZUMAB

A humanized monoclonal immunoglobulin (Ig) G4 antibody directed against human cell surface receptor PD-1 (programmed death-1 or programmed cell death-1) with potential immune checkpoint inhibitory and antineoplastic activities. Upon administration, pembrolizumab binds to PD-1, an inhibitory signaling receptor expressed on the surface of activated T cells, and blocks the binding to and activation of PD-1 by its ligands, which results in the activation of T-cell-mediated immune responses against tumor cells. The ligands for PD-1 include programmed cell death ligand 1 (PD-L1), overexpressed on certain cancer cells, and programmed cell death ligand 2 (PD-L2), which is primarily expressed on APCs. Activated PD-1 negatively regulates T-cell activation and plays a key role in in tumor evasion from host immunity.


Celiprolol

A cardioselective beta-1 adrenergic antagonist that has intrinsic symopathomimetic activity. It is used in the management of ANGINA PECTORIS and HYPERTENSION.


Isoxsuprine

A beta-adrenergic agonist that causes direct relaxation of uterine and vascular smooth muscle. Its vasodilating actions are greater on the arteries supplying skeletal muscle than on those supplying skin. It is used in the treatment of peripheral vascular disease and in premature labor.


Aminoglutethimide

An aromatase inhibitor that is used in the treatment of advanced BREAST CANCER.


Secondary malignant neoplasm of female breast


Flibanserin

a serotonin receptor modulator with high affinity for the following serotonin (5-hydroxytryptamine or 5-T) receptors: agonist activity at 5-HT1A and antagonist activity at 5-HT2A. Flibanserin also has moderate antagonist activities at the 5-HT2B, 5-HT2C, and dopamine D4 receptors, used in the treatment of premenopausal women with hypoactive sexual desire disorder


dibotermin alfa

a member of the TGF-beta superfamily and closely related to the osteogenic/bone morphogenetic proteins


intervertebral disc degenerative disorder


SARGRAMOSTIM

a glycosylated form of recombinant GM-CSF; used to treat drug-induced agranulocytosis; amino acid sequence known


oprelvekin

Oprelvekin is a recombinant human Interleukin-11. Interleukin eleven (IL-11) is a thrombopoietic growth factor that directly stimulates the proliferation of hematopoietic stem cells and megakaryocyte progenitor cells and induces megakaryocyte maturation resulting in increased platelet production.


evolocumab

Evolocumab is a human monoclonal IgG2 directed against human proprotein convertase subtilisin kexin 9 (PCSK9). Evolocumab binds to PCSK9 and inhibits circulating PCSK9 from binding to the low density lipoprotein (LDL) receptor (LDLR), preventing PCSK9-mediated LDLR degradation and permitting LDLR to recycle back to the liver cell surface. By inhibiting the binding of PCSK9 to LDLR, evolocumab increases the number of LDLRs available to clear LDL from the blood, thereby lowering LDL-C levels.


Familial hypercholesterolemia - heterozygous


Befunolol

synonym BFE 60 refers to HCl; RN given refers to parent cpd; structure


Rolapitant

An orally bioavailable, centrally-acting, selective, neurokinin 1 receptor (NK1-receptor) antagonist with potential antiemetic activity. Upon oral administration, rolapitant competitively binds to and blocks the activity of the NK1-receptor in the central nervous system, thereby inhibiting the binding of the endogenous ligand, substance P (SP). This may prevent both SP-induced emesis and chemotherapy-induced nausea and vomiting (CINV). The interaction of SP with the NK1-receptor plays a key role in the induction of nausea and vomiting caused by emetogenic cancer chemotherapy. Compared to other NK1-receptor antagonists, rolapitant has both a more rapid onset of action and a much longer half-life.


A thioxanthene neuroleptic that, unlike CHLORPROMAZINE, is claimed to have CNS-activating properties. It is used in the treatment of psychoses although not in excited or manic patients. (From Martindale, The Extra Pharmacopoeia, 30th ed, p595)


Domperidone

A specific blocker of dopamine receptors. It speeds gastrointestinal peristalsis, causes prolactin release, and is used as antiemetic and tool in the study of dopaminergic mechanisms.


Mesoridazine

A phenothiazine antipsychotic with effects similar to CHLORPROMAZINE.


Fluspirilene

A long-acting injectable antipsychotic agent used for chronic schizophrenia.


ALIROCUMAB

antibody to PCSK9 to manage LDL cholesterol


ANAKINRA

A ligand that binds to but fails to activate the INTERLEUKIN 1 RECEPTOR. It plays an inhibitory role in the regulation of INFLAMMATION and FEVER. Several isoforms of the protein exist due to multiple ALTERNATIVE SPLICING of its mRNA. As IL-1 levels are elevated in patients with COVID-19, anakinra is currently being investigated for its potential to combat cytokine release syndrome (CRS) in patients with severely ill COVID-19.


Eliglustat tartrate

recombinant protein for treatment of Fabry disease


TUROCTOCOG ALFA

a recombinant coagulation factor VIII for prevention and treatment of bleeding in haemophilia A


Gadoversetamide

nonionic, linear chelate used for magnetic resonance imaging of brain and spine


agalsidase alfa

genetically engineered, glycosylated human alpha-galactosidase isoenzyme A used in treatment of Fabry disease


Sacubitril

inhibits neprilysin (neutral endopeptidase; NEP) via LBQ657, the active metabolite and leads to increased levels of peptides that are degraded by neprilysin such as natriuretic peptides. It has been approved in combination with valsartan (under the name ENTRESTO) for the treatment of heart failure.


Trelagliptin

Trelagliptin is an orally active dipeptidyl peptidase (DPP)-4 inhibitor developed by Takeda and approved in Japan for the treatment of type 2 diabetes mellitus.


Pirenzepine

An antimuscarinic agent that inhibits gastric secretion at lower doses than are required to affect gastrointestinal motility, salivary, central nervous system, cardiovascular, ocular, and urinary function. It promotes the healing of duodenal ulcers and due to its cytoprotective action is beneficial in the prevention of duodenal ulcer recurrence. It also potentiates the effect of other antiulcer agents such as CIMETIDINE and RANITIDINE. It is generally well tolerated by patients.


Neridronic acid

used for therapy of Paget's disease of bone & malignant hypercalcaemia


Incadronic acid

increases serum 1,25-dihydroxyvitamin D in rats via stimulating renal 1-hydroxylase activity; structure given in first source


Brexpiprazole

an atypical antipsychotic the efficacy of brexpiprazole may be mediated through a combination of partial agonist activity at serotonin 5-HT1A and dopamine D2 receptors, and antagonist activity at serotonin 5-HT2A receptors


pegvisomant

is a genetically engineered analogue of human growth hormone that functions as a growth hormone-receptor antagonist; used to treat Acromegaly


velaglucerase alfa

produced by gene activation technology in a human fibroblast cell line and has the same amino acid sequence as the naturally occurring human enzyme glucocerebrosidase


Orphenadrine

A muscarinic antagonist used to treat drug-induced parkinsonism and to relieve pain from muscle spasm.


trastuzumab emtansine

Immunotoxin that consists of humanized monoclonal anti-HER2 antibody TRASTUZUMAB covalently linked to anti-microtubule agent MAYTANSINOID DM1 for treatment of metastatic breast cancer in patients who previously received trastuzumab and a TAXANES, separately or in combination.


Lipegfilgrastim

A long acting glyco-pegylated recombinant form of human granulocyte colony-stimulating factor (G-CSF), with hematopoietic activity. Similar to G-CSF, lipegfilgrastim binds to and activates specific cell surface receptors, and stimulates neutrophil progenitor proliferation and differentiation. Therefore, this agent may prevent the duration and incidence of chemotherapy-induced neutropenia. Compared to filgrastim, lipegfilgrastim has a prolonged plasma half-life.


Moxifloxacin

A fluoroquinolone that acts as an inhibitor of DNA TOPOISOMERASE II and is used as a broad-spectrum antibacterial agent.


RASBURICASE

a recombinant form of enzyme that degrades uric acid to allantoin just downstream of xanthine oxidase; used to treat hyperuricaemia


Mixture of antifungal heptaene macrolides from Streptomyces griseus or Actinomyces levoris used topically in candidiasis. The antibiotic complex is composed of candicidins A, B, C, and D, of which D is the major component.


somatropin

A 191-amino acid polypeptide hormone secreted by the human adenohypophysis (PITUITARY GLAND, ANTERIOR), also known as GH or somatotropin. Synthetic growth hormone, termed somatropin, has replaced the natural form in therapeutic usage such as treatment of dwarfism in children with growth hormone deficiency.


Erythropoietin

Glycoprotein hormone, secreted chiefly by the KIDNEY in the adult and the LIVER in the FETUS, that acts on erythroid stem cells of the BONE MARROW to stimulate proliferation and differentiation.


romiplostim

Romiplostim, a member of the TPO mimetic class, is an Fc-peptide fusion protein (peptibody) that activates intracellular transcriptional pathways leading to increased platelet production via the TPO receptor (also known as cMpl). The peptibody molecule contains two identical single-chain subunits, each consisting of human immunoglobulin IgG1 Fc domain, covalently linked at the C-terminus to a peptide containing two thrombopoietin receptor-binding domains. Romiplostim has no amino acid sequence homology to endogenous TPO. Romiplostim is produced by recombinant DNA technology in Escherichia coli.


laronidase

Laronidase is a polymorphic variant of the human enzyme alpha‑l‑iduronidase that is produced by recombinant DNA technology which catalyzes the hydrolysis of terminal alpha-l-iduronic acid residues of dermatan sulfate and heparan sulfate. Reduced or absent alpha-L-iduronidase activity results in the accumulation of the glycosaminoglycans (GAG) substrates, dermatan sulfate and heparan sulfate, throughout the body and leads to widespread cellular, tissue, and organ dysfunction.


aluminum acetate

astringent for temporary relief of minor skin irritations


ELOSULFASE ALFA

BMN-100 and BMN 110 are recombinant GALNS; RefSeq NM_000512


Gastro-esophageal reflux disease with esophagitis


Xipamide

A sulfamoylbenzamide analog of CLOPAMIDE. It is diuretic and saluretic with antihypertensive activity. It is bound to PLASMA PROTEINS, thus has a delayed onset and prolonged action.


Lumiracoxib

A selective non-steroidal anti-inflammatory drug (NSAID) that belongs to the group cyclo-oxygenase 2 (COX-2) inhibitors. It blocks the COX-2 enzyme, resulting in a reduction in the production of prostaglandins, substances that are involved in the inflammation process.


Azapropazone

An anti-inflammatory agent used in the treatment of rheumatoid arthritis. It also has uricosuric properties and has been used to treat gout.


pegfilgrastim

pegylated (r-G-CSF) conjugated to polyethylene glycol for treatment of neutropenic patients with non-myeloid malignancies who are at high risk for infection


Filgrastim

A recombinant granulocyte colony-stimulating factor (G-CSF) that is used in the treatment and prevention of NEUTROPENIA, preparation and collection of blood progenitor cells, and for use in PERIPHERAL BLOOD STEM CELL TRANSPLANTATION.


Flufenamic acid

An anthranilic acid derivative with analgesic, anti-inflammatory, and antipyretic properties. It is used in musculoskeletal and joint disorders and administered by mouth and topically. (From Martindale, The Extra Pharmacopoeia, 30th ed, p16)


Poldine

major descriptor (66-86); on-line search BENZILATES (66-86); INDEX MEDICUS search POLDINE (66-86); RN given refers to parent cpd


Ivabradine

Ivabradine is a pure heart rate lowering agent, acting by selective and specific inhibition of the cardiac pacemaker If current that controls the spontaneous diastolic depolarisation in the sinus node and regulates heart rate. The cardiac effects are specific to the sinus node with no effect on intra-atrial, atrioventricular or intraventricular conduction times, nor on myocardial contractility or ventricular repolarisation.


Triamcinolone hexacetonide

synthetic glucocorticoid analog used for  anti-inflammatory effects in disorders of many organ systems


SILTUXIMAB

a chimeric anti-interleukin-6 antibody for treatment of multiple myeloma and renal cell carcinoma; in phase I and phase II clinical trials. It was investigated for its potential to combat cytokine release syndrome (CRS) in patients with severely ill COVID-19 but the efficacy and safety of siltuximab in the treatment of COVID-19 were not established.


Amfetaminil

a supposedly insoluble amphetamine complex that may release amphetamine; proposed as central nervous system stimulant; minor desriptor (78-86); on line & INDEX MEDICUS search AMPHETAMINES (75-86); RN given refers to parent compound


etanercept

A recombinant version of soluble human TNF receptor fused to an IgG FC fragment that binds specifically to TUMOR NECROSIS FACTOR and inhibits its binding with endogenous TNF receptors. It prevents the inflammatory effect of TNF and is used to treat RHEUMATOID ARTHRITIS; PSORIATIC ARTHRITIS and ANKYLOSING SPONDYLITIS.


Severe chronic obstructive pulmonary disease


TRASTUZUMAB

The HER2 (or c-erbB2) proto-oncogene encodes a transmembrane receptor protein of 185 kDa, which is structurally related to the epidermal growth factor receptor. Trastuzumab products have been shown, in both in vitro assays and in animals, to inhibit the proliferation of human tumor cells that overexpress HER2. Trastuzumab products are mediators of antibody-dependent cellular cytotoxicity (ADCC). In vitro, trastuzumab product-mediated ADCC has been shown to be preferentially exerted on HER2 overexpressing cancer cells compared with cancer cells that do not overexpress HER2.


Palbociclib

Palbociclib is an inhibitor of cyclin-dependent kinases (CDK) 4 and 6. Cyclin D1 and CDK4/6 are downstream of signaling pathways which lead to cellular proliferation. In vitro, palbociclib reduced cellular proliferation of estrogen receptor (ER)-positive breast cancer cell lines by blocking progression of the cell from G1 into S phase of the cell cycle. Treatment of breast cancer cell lines with the combination of palbociclib and antiestrogens leads to decreased retinoblastoma (Rb) protein phosphorylation resulting in reduced E2F expression and signaling, and increased growth arrest compared to treatment with each drug alone. In vitro treatment of ER-positive breast cancer cell lines with the combination of palbociclib and antiestrogens led to increased cell senescence compared to each drug alone, which was sustained for up to 6 days following palbociclib removal and was greater if antiestrogen treatment was continued. In vivo studies using a patient-derived ER-positive breast cancer xenograft model demonstrated that the combination of palbociclib and letrozole increased the inhibition of Rb phosphorylation, downstream signaling, and tumor growth compared to each drug alone.


Arformoterol

the (R,R)-enantiomer of formoterol, is a selective long-acting beta2-adrenergic receptor agonist (beta2-agonist) that has two-fold greater potency than racemic formoterol


Apremilast

Apremilast is an oral small-molecule inhibitor of phosphodiesterase 4 (PDE4) specific for cyclic adenosine monophosphate (cAMP). PDE4 inhibition results in increased intracellular cAMP levels. The specific mechanism(s) by which apremilast exerts its therapeutic action in psoriatic arthritis patients and psoriasis patients is not well defined.


Tenofovir alafenamide

a phosphonoamidate prodrug of tenofovir (2'deoxyadenosine monophosphate analog). Plasma exposure to tenofovir alafenamide allows for permeation into cells and then tenofovir alafenamide is intracellularly converted to tenofovir through hydrolysis by cathepsin A. Tenofovir is subsequently phosphorylated by cellular kinases to the active metabolite tenofovir diphosphate. Tenofovir diphosphate inhibits HIV replication through incorporation into viral DNA by the HIV reverse transcriptase, which results in DNA chain-termination


Panobinostat

An indole and hydroxamic acid derivative that acts as a HISTONE DEACETYLASE inhibitor. It is used as an antineoplastic agent in combination with BORTEZOMIB and DEXAMETHASONE for the treatment of MULTIPLE MYELOMA.


Avibactam

a non-beta-lactam beta-lactamase inhibitor that inactivates some beta-lactamases and protects ceftazidime from degradation by certain beta-lactamases


Febuxostat

A thiazole derivative and inhibitor of XANTHINE OXIDASE that is used for the treatment of HYPERURICEMIA in patients with chronic GOUT.


Tolvaptan

A benzazepine derivative and selective VASOPRESSIN V2 RECEPTOR antagonist that is used to treat euvolemic and hypervolemic HYPONATREMIA. It is also used in the treatment of rapidly progressing AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE to slow the rate of cyst development and renal insufficiency.


autosomal dominant polycystic kidney disease


Aminolevulinic acid

A compound produced from succinyl-CoA and GLYCINE as an intermediate in heme synthesis. It is used as a PHOTOCHEMOTHERAPY for actinic KERATOSIS.


Roxatidine acetate

a histamine H2 receptor antagonist drug used to treat gastric ulcers


idursulfase

An enzyme that specifically cleaves the ester sulfate of iduronic acid. Its deficiency has been demonstrated in Hunter's syndrome, which is characterized by an excess of dermatan sulfate and heparan sulfate. EC 3.1.6.13.


Benzydamine

A benzyl-indazole having analgesic, antipyretic, and anti-inflammatory effects. It is used to reduce post-surgical and post-traumatic pain and edema and to promote healing. It is also used topically in treatment of RHEUMATIC DISEASES and INFLAMMATION of the mouth and throat.


Finafloxacin

Finafloxacin belongs to the fluoroquinolone class of antibacterials which involves the inhibition of bacterial type II topoisomerase enzymes, DNA gyrase and topoisomerase IV, which are required for bacterial DNA replication, transcription, repair and recombination.


Beraprost

stable prostacyclin analog; structure given in first source


Safinamide

Safinamide acts through both dopaminergic and non-dopaminergic mechanisms of action. Safinamide is a highly selective and reversible MAO-B inhibitor causing an increase in extracellular levels of dopamine in the striatum. Safinamide is associated with state-dependent inhibition of voltage-gated sodium (Na+) channels, and modulation of stimulated release of glutamate. To what extent the nondopaminergic effects contribute to the overall effect has not been established.


Ceftolozane belongs to the cephalosporin class of antibacterial drugs. The bactericidal action of ceftolozane results from inhibition of cell wall biosynthesis, and is mediated through binding to penicillin-binding proteins (PBPs). Ceftolozane is an inhibitor of PBPs of P. aeruginosa (e.g., PBP1b, PBP1c, and PBP3) and E. coli (e.g., PBP3).


Liraglutide

An analog of GLUCAGON-LIKE PEPTIDE 1 and agonist of the GLUCAGON-LIKE PEPTIDE 1 RECEPTOR that is used as a HYPOGLYCEMIC AGENT and supplemental therapy in the treatment of DIABETES MELLITUS by patients who do not respond to METFORMIN.


Tibolone

used in prevention of postmenopausal osteoporosis


Rimonabant

A pyrazole and piperidine derivative that acts as a selective cannabinoid type-1 receptor (CB1 RECEPTOR) antagonist. It inhibits the proliferation and maturation of ADIPOCYTES, improves lipid and glucose metabolism, and regulates food intake and energy balance. It is used in the management of OBESITY.


Reboxetine

A morpholine derivative that is a selective and potent noradrenaline reuptake inhibitor; it is used in the treatment of DEPRESSIVE DISORDER.


Bezafibrate

An antilipemic agent that lowers CHOLESTEROL and TRIGLYCERIDES. It decreases LOW DENSITY LIPOPROTEINS and increases HIGH DENSITY LIPOPROTEINS.


Cosyntropin

A synthetic peptide that is identical to the 24-amino acid segment at the N-terminal of ADRENOCORTICOTROPIC HORMONE. ACTH (1-24), a segment similar in all species, contains the biological activity that stimulates production of CORTICOSTEROIDS in the ADRENAL CORTEX.


Tienilic acid

a novel diuretic with uricosuric action, it has been used as an antihypertensive agent, withdrawn shortly after approval due to liver toxicity


Lutein

A xanthophyll found in the major LIGHT-HARVESTING PROTEIN COMPLEXES of plants. Dietary lutein accumulates in the MACULA LUTEA.


BLINATUMOMAB

a single-chain bispecific recombinant antibody from Micromet's BiTE (bispecific T-cell engager) product platform that binds both the CD19 antigen and the T-cell receptor (CD3), for the potential treatment of B-cell lymphoma


Lidocaine

A local anesthetic and cardiac depressant used as an antiarrhythmia agent. Its actions are more intense and its effects more prolonged than those of PROCAINE but its duration of action is shorter than that of BUPIVACAINE or PRILOCAINE.


Prilocaine

A local anesthetic that is similar pharmacologically to LIDOCAINE. Currently, it is used most often for infiltration anesthesia in dentistry.


AFLIBERCEPT

has antiangiogenic activity; consists of the second and third Ig domains of VEGFR1 and VEGFR2, respectively, fusion to the Fc region of IgG1; putative antineoplastic agent that binds all VEGF-A isoforms; possible medicine for eye disorders


central retinal vein occlusion with macular edema


Branch retinal vein occlusion with macular edema


metastasis from malignant tumor of colon


Netupitant

Netupitant is a selective antagonist of human substance P/neurokinin 1 (NK-1) receptors. Delayed emesis has been largely associated with the activation of tachykinin family neurokinin 1 (NK-1) receptors (broadly distributed in the central and peripheral nervous systems) by substance P. As shown in in vitro and in vivo studies, netupitant inhibits substance P mediated responses.


Ledipasvir

an inhibitor of the HCV NS5A protein, which is required for viral replication


Sepsis caused by Streptococcus pneumoniae


INSULIN GLARGINE

A recombinant LONG ACTING INSULIN and HYPOGLYCEMIC AGENT that is used to manage BLOOD GLUCOSE in patients with DIABETES MELLITUS.


Lafutidine

histamine H(2)-receptor antagonist used as an antiulcer drug


Duloxetine

A thiophene derivative and selective NEUROTRANSMITTER UPTAKE INHIBITOR for SEROTONIN and NORADRENALINE (SNRI). It is an ANTIDEPRESSIVE AGENT and ANXIOLYTIC, and is also used for the treatment of pain in patients with DIABETES MELLITUS and FIBROMYALGIA.


Benznidazole

Benznidazole inhibits the synthesis of DNA, RNA, and proteins within the T. cruzi parasite. Studies suggest that benznidazole is reduced by a Type I nitroreductase (NTR) enzyme of T. cruzi producing a series of short-lived intermediates that may promote damage to several macromolecules including DNA. In mammalian cells, however, benznidazole is metabolized by reduction of the nitro group to an amino group by a Type II NTR enzyme. The precise mechanism of action is not known. Benznidazole is active against all three stages, trypomastigotes, amastigotes, and epimastigotes, of T. cruzi. However, the sensitivity of T. cruzi strains to benznidazole, from different geographic regions, may vary.


Diethylcarbamazine

An anthelmintic used primarily as the citrate in the treatment of filariasis, particularly infestations with Wucheria bancrofti or Loa loa.


Eflornithine

An inhibitor of ORNITHINE DECARBOXYLASE, the rate limiting enzyme of the polyamine biosynthetic pathway.


Melarsoprol

Arsenical used in trypanosomiases. It may cause fatal encephalopathy and other undesirable side effects.


Nifurtimox

A nitrofuran thiazine that has been used against TRYPANOSOMIASIS.


Antimony sodium gluconate

Antimony complex where the metal may exist in either the pentavalent or trivalent states. The pentavalent gluconate is used in leishmaniasis. The trivalent gluconate is most frequently used in schistosomiasis.


Suramin

A polyanionic compound with an unknown mechanism of action. It is used parenterally in the treatment of African trypanosomiasis and it has been used clinically with diethylcarbamazine to kill the adult Onchocerca. (From AMA Drug Evaluations Annual, 1992, p1643) It has also been shown to have potent antineoplastic properties.


Pentamidine

Antiprotozoal agent effective in trypanosomiasis, leishmaniasis, and some fungal infections; used in treatment of PNEUMOCYSTIS pneumonia in HIV-infected patients. It may cause diabetes mellitus, central nervous system damage, and other toxic effects.


dulaglutide

a glucagon-like peptide-1 receptor agonist; GLP-1 immunoglobulin G (IgG4) Fc fusion protein with extended activity


Beclomethasone dipropionate

An anti-inflammatory, synthetic glucocorticoid. It is used topically as an anti-inflammatory agent and in aerosol form for the treatment of ASTHMA.


Cilazapril

One of the ANGIOTENSIN-CONVERTING ENZYME INHIBITORS (ACE inhibitors) used for hypertension. It is a prodrug that is hydrolyzed after absorption to its main metabolite cilazaprilat.


Cetilistat

lipase inhibitor in randomized, placebo-controlled study of weight reduction in obese patients (3/2007)


Trichlormethiazide

A thiazide diuretic with properties similar to those of HYDROCHLOROTHIAZIDE. (From Martindale, The Extra Pharmacopoeia, 30th ed, p830)


calfactant

Infasurf is tradename; a lung surfactant extract from new born calves containing 35 mg/mL phospholipid & 2% surfactant-associated proteins B & C (wt/wt)


Cobicistat

A carbamate and thiazole derivative that functions as a CYTOCHROME P450 CYP3A INHIBITOR to enhance the concentration of ANTI-HIV AGENTS, with which it is used in combination, for the treatment of HIV INFECTIONS.


Enoxaparin sodium

Low-molecular-weight fragment of heparin, having a 4-enopyranosuronate sodium structure at the non-reducing end of the chain. It is prepared by depolymerization of the benzylic ester of porcine mucosal heparin. Therapeutically, it is used as an antithrombotic agent. (From Merck Index, 11th ed)


glatiramer acetate

A random polymer of L-ALANINE, L-GLUTAMIC ACID, L-LYSINE, and L-TYROSINE that structurally resembles MYELIN BASIC PROTEIN. It is used in the treatment of RELAPSING-REMITTING MULTIPLE SCLEROSIS.


Cyclic basic peptide related to VIOMYCIN. It is isolated from an induced mutant of Streptomyces griseoverticillatus var. tuberacticus and acts as an antitubercular agent with less ototoxicity than tuberactinomycin.


Suvorexant

a highly selective antagonist for orexin receptors OX1R and OX2R, the orexin neuropeptide signaling system is a central promoter of wakefulness, blocking the binding of wake-promoting neuropeptides orexin A and orexin B to receptors OX1R and OX2R is thought to suppress wake drive


Dexetimide

A muscarinic antagonist that has been used to treat neuroleptic-induced parkinsonism. Benzetimide is the (-)-enantimorph of dexetimide.


Eliglustat

Eliglustat tartrate is a small molecule inhibitor of glucosylceramide synthase that resembles the ceramide substrate for the enzyme. Gaucher disease is caused by a deficiency of the lysosomal enzyme acid beta-glucosidase. Acid beta-glucosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency causes an accumulation of glucosylceramide (GL-1) primarily in the lysosomal compartment of macrophages, giving rise to foam cells or "Gaucher cells".


Istradefylline

selective antagonist at the A2A receptor, used in the treatment of Parkinson's disease


Efinaconazole

Efinaconazole is an azole antifungal. Efinaconazole inhibits fungal lanosterol 14α-demethylase involved in the biosynthesis of ergosterol, a constituent of fungal cell membranes.