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Displaying drugs 376 - 400 of 1450 in total
Tofersen is under an intrathecally administered antisense oligonucleotide targeting the mutated SOD1 gene that causes amyotrophic lateral sclerosis (ALS). Although there were various causes of ALS, 2% of ALS cases are due to SOD1 mutations, with more than 200 SOD1 mutations documented.[A259028,A259033] Tofersen was granted accelerated approval from the FDA...
Approved
Investigational
Abrocitinib is an oral small-molecule inhibitor of Janus kinase 1 (JAK1). Janus kinases are intracellular enzymes involved in transduction pathways that regulate hematopoiesis and immune cell function. The Janus kinase (JAK)–signal transducer and activator of transcription (STAT) signalling pathway plays a central role in the pathogenesis of a variety of...
Approved
Investigational
Pemigatinib is a small molecule kinase inhibitor with antitumour activity. It works by inhibiting fibroblast growth factor receptors (FGFRs), which are receptor tyrosine kinases that activate signalling pathways in tumour cells. FGFRs gained attention as potential therapeutic targets in selected cancers, as FGFR gene alterations were observed in a wide...
Approved
Investigational
Calcium carbimide, sold as the citrate salt, is an alcohol-sensitizing agent. Its effects are similar to the drug disulfiram (Antabuse) in that it interferes with the normal metabolism of alcohol by preventing the breakdown of the metabolic product acetaldehyde. Calcium carbimide was conceived as an alternative for the treatment of...
Approved
Withdrawn
Lorlatinib is a third-generation ALK tyrosine kinase inhibitor (TKI) for patients with ALK-positive metastatic non-small cell lung cancer which was first approved by the US FDA in November of 2018. It was subsequently approved by the EMA in 2019 for the treatment of select patients with previously treated advanced ALK-positive...
Approved
Investigational
Matched Iupac: … (16R)-19-amino-13-fluoro-4,8,16-trimethyl-9-oxo-17-oxa-4,5,8,20-tetraazatetracyclo[16.3.1.0^{2,6}.0^{ …
Retifanlimab is a humanized IgG4 kappa monoclonal antibody that binds to the programmed death receptor-1 (PD-1), blocking PD-1 interaction with its ligands, programmed death-ligand 1 (PD-L1) and programmed death-ligand 2 (PD-L2). By blocking the PD-1/PD-L1/2 pathway, retifanlimab potentiates T-cell activity and boosts the immune response against cancer cells. Other monoclonal...
Approved
Investigational
Approved
Matched Mixtures name: … Phoxillum B22k4/0
Bumetanide is a sulfamyl diuretic.
Approved
Multiple sclerosis, or MS, is a devastating inflammatory disease that often progresses and causes severe neurological, physical, and cognitive effects. Fingolimod is a sphingosine 1-phosphate receptor modulator for the treatment of relapsing-remitting multiple sclerosis. It was developed by Novartis and initially approved by the FDA in 2010. Fingolimod was also...
Approved
Investigational
Matched Salts cas: … 162359-56-0
Approved
Investigational
Matched Iupac: … {1,26}.0^{3,8}.0^{23,27}.0^{25,42}.0^{32,44}.0^{39,43}.0^{37,45}]heptatetraconta-2(47),3,5,7,27,29(44 ... 2lambda5,9,19,26,43lambda5,44lambda5,45lambda5-heptaaza-15lambda5-phospha-1-cobaltadodecacyclo[27.14.1.1^{1,34}.1^{2,9}.1^{10,13}.0^ …
Fostemsavir is the phosphonooxymethyl prodrug of temsavir, a novel HIV-1 attachment inhibitor. It binds to and inhibits the activity of gp120, a subunit within the HIV-1 gp160 envelope glycoprotein that facilitates the attachment of HIV-1 to host cell CD4 receptors - in doing so, temsavir prevents the first step in...
Approved
Investigational
A 9,10alpha-dihydro derivative of ergotamine. Dihydroergotamine is used as an abortive therapy for migraines. Its use has largely been supplanted by triptans in current therapy due to the class's greater selectivity and more favourable side effect profile. Recent improvements have been made in the design of intranasal delivery devices allowing...
Approved
Investigational
Matched Iupac: … dioxo-3-oxa-6,9-diazatricyclo[7.3.0.0^{2,6}]dodecan-4-yl]-6-methyl-6,11-diazatetracyclo[7.6.1.0^{2,7}.0^ …
Fesoterodine is an antimuscarinic prodrug for the treatment of overactive bladder syndrome.
Approved
Lurbinectedin is a DNA alkylating agent that has been investigated in the treatment of a variety of cancers, including mesothelioma, chronic lymphocytic leukemia (CLL), breast cancer, and small-cell lung cancer (SCLC). It is a derivative of the marine-derived agent ecteinascidin (trabectedin), an anticancer agent found in extracts of the tunicate...
Approved
Investigational
Matched Iupac: … {2,13}.0^{4,9}.0^{15,23}.0^{16,20}]triacontane-26,1'-pyrido[3,4-b]indole]-4,6,8,15,20,22-hexaen-22-yl ... trimethyl-27-oxo-2',3',4',9'-tetrahydro-17,19,28-trioxa-24-thia-13,30-diazaspiro[heptacyclo[12.9.6.1^{3,11}.0^ …
Buprenorphine is a weak partial mu-opioid receptor agonist and a weak kappa-opioid receptor antagonist used for the treatment of severe pain.[A186283,A186292] It is also commonly used as an alternative to methadone for the treatment of severe opioid addiction. Buprenorphine is commercially available as the brand name product Suboxone which is...
Approved
Illicit
Investigational
Vet approved
Matched Iupac: … {1,6}.0^{6,14}.0^{7,12}]icosa-7,9,11-trien-11-ol ... cyclopropylmethyl)-16-[(2S)-2-hydroxy-3,3-dimethylbutan-2-yl]-15-methoxy-13-oxa-3-azahexacyclo[13.2.2.1^{2,8}.0^ …
Mupirocin, formerly termed pseudomonic acid A, is a novel antibacterial agent with a unique chemical structure and mode of action apart from other antibiotic agents. Produced by fermentation using the organism Pseudomonas fluorescens, mupirocin is a naturally-occurring antibiotic that displays a broad-specturm activity against many gram-positive bacteria and certain gram-negative...
Approved
Investigational
Vet approved
Vutrisiran is a double-stranded small interfering ribonucleic acid (siRNA) that targets wild-type and mutant transthyretin (TTR) messenger RNA (mRNA). This siRNA therapeutic is indicated for the treatment of neuropathies associated with hereditary transthyretin-mediated amyloidosis (ATTR), a condition caused by mutations in the TTR gene. More than 130 TTR mutations have...
Approved
Investigational
Nalmefene, a 6-methylene analogue of naltrexone, is an opioid receptor antagonist. It acts as an antagonist at the mu (μ)-opioid and delta (δ)-opioid receptors and a partial agonist at the kappa (κ)-opioid receptor. In Europe, nalmefene oral tablets are used to reduce alcohol consumption in adults with alcohol dependence. Nalmefene...
Approved
Investigational
Withdrawn
Matched Iupac: … (1S,5R,13S,17S)-4-(cyclopropylmethyl)-14-methylidene-12-oxa-4-azapentacyclo[9.6.1.0^{1,13}.0^{5,17}.0
Matched Salts cas: … 58895-64-0
Gepirone, an azapirone, is a pharmacologic analog of buspirone that acts selectively on the pre- and post-synaptic 5HT1A receptors. Although earlier clinical trials showed promising results for gepirone, its formulation as an immediate-release tablet necessitates frequent administration due to the short half-lives. It was not until an extended-release formulation of...
Approved
Investigational
An opioid analgesic with actions and uses similar to those of morphine, apart from an absence of cough suppressant activity. It is used in the treatment of moderate to severe pain, including pain in obstetrics. It may also be used as an adjunct to anesthesia (From Martindale, The Extra Pharmacopoeia,...
Approved
Investigational
Vet approved
Matched Iupac: … (1S,5R,13R,17S)-10,17-dihydroxy-4-methyl-12-oxa-4-azapentacyclo[9.6.1.0^{1,13}.0^{5,17}.0^{7,18}]octadeca …
Tebentafusp is a gp100 peptide-HLA-directed CD3 T cell engager. It is a bispecific, fusion protein and first-in-class drug of immune-mobilizing monoclonal T cell receptors against cancer (ImmTACs), a recently developed cancer immunotherapy with a novel mechanism of action. ImmTACs bind to target cancer cells that express a specific antigen of...
Approved
Investigational
The relief of pain (analgesia) is a primary goal for enhancing the quality of life of patients and for increasing the ability of patients to engage in day to day activities. Codeine, an opioid analgesic, was originally approved in the US in 1950 and is a drug used to decrease...
Approved
Illicit
Matched Iupac: … (1S,5R,13R,14S,17R)-10-methoxy-4-methyl-12-oxa-4-azapentacyclo[9.6.1.0^{1,13}.0^{5,17}.0^{7,18}]octadeca …
Mavorixafor is a CXC chemokine receptor 4 (CXCR4) antagonist. It was first approved by the FDA on April 30, 2024, for the treatment of warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome, a genetic immunodeficiency disorder characterized by a reduced number of mature neutrophils and lymphocytes. WHIM syndrome is caused by...
Approved
Investigational
CD20, an antigen expressed by various B and T cells, is an attractive therapeutic target in various cancers and autoimmune conditions.[A244195, A244200, A244205] Monoclonal antibodies for B cell depletion rely on their binding affinity and ability to clear bound cells through mechanisms such as antibody-dependent cellular cytotoxicity (ADCC), mediated by...
Approved
Investigational
Matched Description: … [A244210] Ublituximab is a chimeric anti-CD20 IgG1κ antibody produced in the rat YB2/0 cell line to target …
Trimetazidine is a piperazine derivative indicated for the symptomatic treatment of stable angina pectoris in patients inadequately controlled or intolerant to first line therapies. Trimetazidine has been studied as a treatment for angina pectoris since the late 1960s.[A233255,A233260] Acidic conditions, caused by anaerobic metabolism and fatty acid oxidation, in response...
Approved
Investigational
Matched Salts cas: … 13171-25-0
Displaying drugs 376 - 400 of 1450 in total