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Displaying results 841 - 850 of 4575 in total
  1. Drug

    Tezacaftor

    go.drugbank.com/drugs/DB11712
    ApprovedInvestigational

    [L1595] It was developed by Vertex Pharmaceuticals and FDA approved in combination with [ivacaftor] to manage cystic fibrosis.[L6814] This drug was approved by the FDA on February 12, 2018. … [L4894] Cystic Fibrosis is an autosomal recessive disorder caused by one of several different mutations in the gene for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, an ion

  2. Drug

    Mavorixafor

    go.drugbank.com/drugs/DB05501
    ApprovedInvestigational

    by a reduced number of mature neutrophils and lymphocytes. … [L50647] WHIM syndrome is caused by mutations in the _CXCR4_ gene, which leads to overactivation of CXCR4 signalling pathways.[A263652] Mavorixafor prevents the activation of CXCR4.

  3. Drug

    Desipramine

    go.drugbank.com/drugs/DB01151
    Approved

    In non-depressed individuals, desipramine does not affect mood or arousal, but may cause sedation. In depressed individuals, desipramine exerts a positive effect on mood.

  4. Drug

    Remestemcel-L

    go.drugbank.com/drugs/DB13973
    ApprovedInvestigationalWithdrawn

    [L11022] Remestemcel-L was later approved by the FDA on December 18, 2024. … of the recipients [A31818]. aGvHD is often characterized by the presence of inflammatory cascades through signalling of activated T cells.

  5. Drug

    Lanadelumab

    go.drugbank.com/drugs/DB14597
    ApprovedInvestigational

    [A38676] Lanadelumab was approved for use in patients with hereditary angioedema by the FDA in August 2018,[L4537] followed by Health Canada in October 2018[L49226] and the EMA in November 2018.

  6. Drug

    Orforglipron

    go.drugbank.com/drugs/DB18964
    ApprovedInvestigational

    [A275476] Developed to circumvent the adherence barriers and strict administration requirements of peptide-based GLP-1 therapies, its small-molecule structure enables once-daily oral dosing without food

  7. Drug

    Adagrasib

    go.drugbank.com/drugs/DB15568
    ApprovedInvestigational

    The phase II of the same study showed that in patients with KRAS<sup>G12C</sup>-mutated non-small-cell lung cancer (NSCLC), adagrasib was efficient without new safety signals. … Adagrasib (MRTX849) is an oral, small-molecule KRAS inhibitor developed by Mirati Therapeutics.

  8. Drug

    Paliperidone

    go.drugbank.com/drugs/DB01267
    ApprovedInvestigational

    The mechanism of action is unknown but it is likely to act via a similar pathway to risperidone. … Paliperidone was approved by the FDA for treatment of schizophrenia on December 20, 2006.

  9. Drug

    Human C1-esterase inhibitor

    go.drugbank.com/drugs/DB06404
    ApprovedInvestigational

    The disease is characterized by acute attacks of painful, and in some cases, fatal swelling of several soft tissues or edema, which may last up to five days when untreated.[L16586, L16606] … [L16586, L16606] This drug is indicated for prophylaxis and treatment of Hereditary Angioedema (HAE), a human genetic disorder caused by a shortage of C1 inhibitor activity that results in an overreaction

  10. Drug

    Etranacogene dezaparvovec

    go.drugbank.com/drugs/DB16791
    ApprovedInvestigational

    [L45255] Etranacogene dezaparvovec was approved by the EMA in February 2023[L45439,L45444] and by Health Canada in October 2023.[L48801] … [L44156] Etranacogene dezaparvovec was approved by the FDA in November 2022 for the treatment of select patients with hemophilia B, becoming the first gene therapy approved for this indication.