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Displaying results 1901 - 1910 of 10000 in total
  1. Drug

    Danicopan

    go.drugbank.com/drugs/DB15401
    ApprovedInvestigational

    [A263506] It was subsequently approved by the FDA in March 2024.[L50386] … [L50381] It was first approved in January 2024 in Japan for patients with PNH,[A263506,L50416] shortly after which the EMA adopted a positive opinion and recommended granting it marketing authorization

    Synonyms: (2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridin-2-yl)-4-fluoropyrrolidine-2-carboxamide, (2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidine-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridine-2-yl)-4-fluoropyrrolidine-2-carboxamide
  2. Drug

    Efgartigimod alfa

    go.drugbank.com/drugs/DB15270
    ApprovedInvestigational

    [A243759,A243784] Efgartigimod alfa is a first-in-class[L39501] antagonist of the neonatal Fc receptor (FcRn) used in the treatment of MG. … and a reduction in electrical nerve impulses.

  3. Drug

    Cerliponase alfa

    go.drugbank.com/drugs/DB13173
    ApprovedInvestigational

    [L51329] CLN2 is a predominantly pediatric-onset neurodegenerative disease caused by a deficiency in the lysosomal enzyme TPP1, leading to progressive impairment in motor and cognitive function. … [A264354, A264369] As a recombinant human TPP1, cerliponase alfa is used as an enzyme replacement therapy to restore the levels of TPP1 in patients with CLN2.[L51339]

    Categories: Hydrolytic Lysosomal N-terminal Tripeptidyl PeptidaseSynonyms: Immature tripeptidyl-peptidase I
  4. Drug

    Olezarsen

    go.drugbank.com/drugs/DB18728
    ApprovedInvestigational

    Olezarsen is an antisense oligonucleotide that targets the messenger RNA (mRNA) for apolipoprotein C-III (APOC3, apoC-III),[L52033] which is a key regulator of triglyceride levels in plasma, in the liver … [L52033] On December 19, 2024, olezarsen was approved by the FDA for the treatment of familial chylomicronemia syndrome.[L52028] It is also being investigated for the treatment of hyperlipidemia.

  5. Drug

    JPC-3210

    go.drugbank.com/drugs/DB15609
    Experimental

    It is an erythrocytic schizonticide with potent in vitro antimalarial activity against multidrug-resistant _Plasmodium falciparum_ lines, low cytotoxicity, potent in vivo efficacy against murine malaria … JPC-3210 is a potent antimalarial agent.

  6. Drug

    Nitisinone

    go.drugbank.com/drugs/DB00348
    Approved

    Nitisinone is a synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase. It is used in the treatment of hereditary tyrosinemia type 1 and Alkaptonuria. … It is sold under the brand names Orfadin and Harliku. [L53253, L53258]

  7. Drug

    Asciminib

    go.drugbank.com/drugs/DB12597
    ApprovedInvestigational

    [A241065] Asciminib is unique in that it acts as an allosteric inhibitor, binding at the myristoyl pocket of the BCR-ABL1 protein and locking it into an inactive conformation. … More specifically, it is an inhibitor of the ABL1 kinase activity of the BCR-ABL1 fusion protein[L38995] which serves as a driver of CML proliferation in most patients with the disease.

  8. Drug

    Fostamatinib

    go.drugbank.com/drugs/DB12010
    ApprovedInvestigational

    It was approved on April 17, 2018, under the trade name Tavalisse for use in ITP [L2644, L52150]. Fostamatinib has also been granted orphan drug status by the FDA [L2644]. … Recently, fostamatinib has been identified as a potential therapeutic for controlling acute respiratory distress syndrome (ARDS) in patients with severe COVID-19 through its ability to modulate the SYK

  9. Drug

    Mexazolam

    go.drugbank.com/drugs/DB15489
    Investigational

    [A184910] The use of benzodiazepines in the treatment of anxiety is generally a second line measure.[A184910] … [A184910] Mexazolam's structure is similar to that of other benzodiazepines such as [oxazolam] and [cloxazolam].

  10. Drug

    Chlorpropamide

    go.drugbank.com/drugs/DB00672
    ApprovedWithdrawn

    Up to 80% of the single oral dose of chlorpropramide is metabolized, likely in the liver; 80-90% of the dose is excreted in urine as unchanged drug and metabolites. … Medications in this class differ in their dose, rate of absorption, duration of action, route of elimination and binding site on their target pancreatic β cell receptor.

    Synonyms: N-(4-chlorophenylsulfonyl)-N'-propylurea, N-(p-chlorobenzenesulfonyl)-N'-propylureaCategories: Drugs Used in Diabetes