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Alpelisib
go.drugbank.com/drugs/DB12015ApprovedInvestigational[L6652] In April 2022, the FDA granted the use of alpelisib in the treatment of PIK3CA-Related Overgrowth Spectrum (PROS) in adults and children who require systemic therapy.[L41384] … Alpelisib was designed to target this enzyme that appears to be mutated at a rate of nearly 30% in human cancers, leading to hyperactivation.
Tasonermin
go.drugbank.com/drugs/DB11626ApprovedIt was approved for use by the European Medicines Agency in April of 1999 for use as an adjunt to surgery for the subsequent removal of the tumor and in palliative care for irresectable soft tissue sarcoma
Eliglustat
go.drugbank.com/drugs/DB09039ApprovedInvestigational[L41404,A7634] Eliglustat was approved by the FDA in August 2014 as an oral substrate reduction therapy for the first-line treatment of type 1 Gaucher disease. … In patients with Gaucher disease, the accumulation of glucosylceramide leads to the formation of Gaucher cells that infiltrate the liver, spleen, bone marrow and other organs.
Synonyms: N-[(1R,2R)-1-(2,3-Dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(1-pyrrolidinyl)-2-propanyl]octanamideCastor oil
go.drugbank.com/drugs/DB11113ApprovedNutraceuticalVet approvedCastor oil is found in over-the-counter oral liquids as a stimulant laxative, and is also added in commercial cosmetic, hair, and skincare products. … Castor oil does not contain ricin, which is a natural poison found in the castor oil plant; the toxic lectin remains in the bean pulp following oil isolation [F70].
Mixtures: Trypsin ComplexCategories: Complex MixturesEthambutol
go.drugbank.com/drugs/DB00330ApprovedInvestigational[L31743] Ethambutol was first described in the literature in 1961.[A229048] It was developed out of a need for therapies active against isoniazid resistant strains of _Mycobacterium tuberculosis_. … Ethambutol is a bacteriostatic agent indicated alongside medications such as [isoniazid], [rifampin], and [pyrazinamide] in the treatment of pulmonary tuberculosis.
Synonyms: (+)-N,N'-bis(1-(hydroxymethyl)propyl)ethylenediamineProducts: E-BUTOL TABLET 400 mgSuzetrigine
go.drugbank.com/drugs/DB18927ApprovedInvestigational[A264953] On January 30, 2025, suzetrigine was approved by the FDA as a first-in-class non-opioid analgesic.[L52098] … Suzetrigine is a Na<sub>V</sub>1.8 voltage-gated sodium channel blocker with analgesic properties[A264948] Na<sub>V</sub>1.8 is abundantly expressed in peripheral sensory nerves to play a role in pain
Acetohydroxamic acid
go.drugbank.com/drugs/DB00551ApprovedInvestigationalAcetohydroxamic Acid, a synthetic drug derived from hydroxylamine and ethyl acetate, is similar in structure to urea. In the urine, it acts as an antagonist of the bacterial enzyme urease. … It is used, in addition to antibiotics or medical procedures, to treat chronic urea-splitting urinary infections.
Synonyms: N-Hydroxyacetamide, N-AcetylhydroxylamineElexacaftor
go.drugbank.com/drugs/DB15444ApprovedInvestigational[A187361] It received FDA approval in October 2019 in combination with [tezacaftor] and [ivacaftor] as the combination product Trikafta<sup>TM</sup>. … [A187361] While dual corrector/potentiator combination therapy has proven useful in the treatment of a subset of CF patients,[A187358] their use is typically limited to patients who are homozygous for
rsPSMA Vaccine
go.drugbank.com/drugs/DB06361ApprovedWithdrawnIn preclinical studies, rsPSMA elicited high-titer antibodies that strongly cross-reacted with prostate cancer cells. [L1039] … Prostate-specific membrane antigen (PSMA) is an ∼100 kDa transmembrane glycoprotein that is abundantly and preferentially expressed in prostate cancer, including recurrent and hormone-refractory cases.
Mucor racemosus
go.drugbank.com/drugs/DB10808ApprovedMucor racemosus extract is used in allergenic testing.
Mixtures: Number Ten Mold Mixture, Number Ten Mold Mixture