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  1. Drug

    Bempedoic acid

    go.drugbank.com/drugs/DB11936
    ApprovedInvestigational

    [L12180] Bempedoic acid is first-in-class adenosine triphosphate-citrate lyase (ACL) inhibitor used once a day for reducing LDL cholesterol levels in statin-refractory patients. … A combination product of bempedoic acid and [ezetimibe] was approved on February 26, 2020 for increased control of LDL cholesterol levels in patients experiencing refractory elevations despite previous

  2. Drug

    Dapiprazole

    go.drugbank.com/drugs/DB00298
    Approved

    Dapiprazole (U.S. trade name Rev-Eyes) is an alpha blocker. It is found in ophthalmic solutions used to reverse mydriasis after an eye examination.

  3. Drug

    Voxelotor

    go.drugbank.com/drugs/DB14975
    Approved

    [T734] Voxelotor was granted accelerated FDA approval on November 25 2019, as it is likely to be a promising treatment for the 100,000 individuals in the U.S. suffering from the disease, in addition … This is a genetically inherited condition most prevalent in the Middle East, Africa, and certain parts of India.

  4. Drug

    Voretigene neparvovec

    go.drugbank.com/drugs/DB13932
    ApprovedInvestigational

    [A31477] An advantage of this adeno-associated virus is the high predictability, unlike retrovirus, as they associate with a specific region of the human cellular genome localized in the chromosome 19. … When used in genetic therapy, this virus is modified for the elimination of its negligible integrative capacity by the removal of *rep* and *cap* and the insertion of the desired gene with its promoter

    Synonyms: VN-rzyl
  5. Drug

    Adagrasib

    go.drugbank.com/drugs/DB15568
    ApprovedInvestigational

    [A254052] In a phase I/IB clinical study that included patients with KRAS<sup>G12C</sup>-mutated advanced solid tumors (NCT03785249), adagrasib exhibited anti-tumor activity. … KRAS mutations are highly common in cancer and account for approximately 85% of all RAS family mutations.

  6. Drug

    Sulfadoxine

    go.drugbank.com/drugs/DB01299
    ApprovedInvestigationalWithdrawn

    A long acting sulfonamide that is used, usually in combination with other drugs, for respiratory, urinary tract, and malarial infections.

  7. Drug

    Deuruxolitinib

    go.drugbank.com/drugs/DB18847
    ApprovedInvestigational

    [L51083] Alopecia areata is an autoimmune condition that attacks the hair follicles and leads to unpredictable hair loss in the scalp and other areas of the body. … [A264108] The Janus kinase (JAK) signal transducer and activator of transcription (STAT) pathway has been implicated in the pathophysiology of alopecia areata, as it regulates the expression of inflammatory

  8. Drug

    Istradefylline

    go.drugbank.com/drugs/DB11757
    ApprovedInvestigational

    [L8237] This drug was first approved in Japan on 25 March 2013.[A184067] Istradefylline was granted FDA approval on 27 August 2019.[L8213] … [A184067] This region of the brain is highly involved in motor control.[A184067] Istradefylline is indicated as an adjunct treatment to [levodopa] and [carbidopa] for Parkinson's disease.

  9. Drug

    Onasemnogene abeparvovec

    go.drugbank.com/drugs/DB15528
    ApprovedInvestigational

    Onasemnogene abeparvovec is the first gene therapy that was approved for this indication in the USA. … [Nusinersen] is another gene therapy that is currently approved by the FDA for the treatment of SMA in pediatric and adult patients.

  10. Drug

    Clascoterone

    go.drugbank.com/drugs/DB12499
    ApprovedInvestigational

    [A218846] In August 2020, FDA approved clascoterone for the first-in-class topical treatment of acne (acne vulgaris) in male and female patients 12 years and older. … Clascoterone (cortexolone 17α-propionate, CB-03-01) is a novel antagonist of androgen receptors. It binds to androgen receptors with high affinity.