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Flavoxate
go.drugbank.com/drugs/DB01148ApprovedA drug that has been used in various urinary syndromes and as an antispasmodic. Its therapeutic usefulness and its mechanism of action are not clear. … It may have local anesthetic activity and direct relaxing effects on smooth muscle as well as some activity as a muscarinic antagonist. [PubChem]
Osilodrostat
go.drugbank.com/drugs/DB11837ApprovedInvestigational[A191850] Osilodrostat was approved for use in the EU in January 2020 for the treatment of endogenous Cushing's syndrome (i.e. … [A191910] As an orally bioavailable drug therapy, osilodrostat provides a novel treatment option for patients in whom removal of the causative tumor is not an option or for whom previous pituitary surgery
Tazemetostat
go.drugbank.com/drugs/DB12887ApprovedInvestigational[L11476] Tazemetostat was first named in literature as EPZ-6438.[A190363] Tazemetaostat was granted FDA approval on 23 January 2020.[L11476]
Penicillamine
go.drugbank.com/drugs/DB00859ApprovedInvestigationalIt is an α-amino acid metabolite of penicillin, although it has no antibiotic properties.
Categories: Compounds used in a research, industrial, or household settingAtidarsagene autotemcel
go.drugbank.com/drugs/DB17538ApprovedInvestigational[L45191] Libmeldy was granted orphan designation by the EMA in April 2007, and was issued a marketing authorization in the EU in December 2020 for the treatment of certain manifestations of metachromatic … [L45196] It was granted FDA approval under the brand name Lenmeldy in March 2024.[L50256]
Synonyms: Autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) geneBrepocitinib
go.drugbank.com/drugs/DB15003ApprovedInvestigationalBrepocitinib is an oral Janus kinase (JAK) and tyrosine kinase 2 (TYK2) inhibitor used to treat dermatomyositis in adults. … [L64097] The FDA approved brepocitinib on August 27, 2026 for the treatment of dermatomyositis in adult patients, making it the first targeted oral therapy approved for the condition.[L64115]
Fosdenopterin
go.drugbank.com/drugs/DB16628ApprovedIn 2009 a recombinant, E. coli-produced cPMP was granted orphan drug designation by the FDA, becoming the first therapeutic option for patients with MoCD type A. … Molybdenum cofactor deficiency (MoCD) is an exceptionally rare autosomal recessive disorder resulting in a deficiency of three molybdenum-dependent enzymes: sulfite oxidase (SOX), xanthine dehydrogenase
Nerandomilast
go.drugbank.com/drugs/DB18237ApprovedInvestigational[L54126] On October 7, 2025, nerandomilast was approved as the first new treatment for idiopathic pulmonary fibrosis in more than a decade. … [A274581] On December 19, 2025, the U.S. FDA approved nerandomilast tablets for the treatment of progressive pulmonary fibrosis (PPF) in adults.
Suzetrigine
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
Olutasidenib
go.drugbank.com/drugs/DB16267ApprovedInvestigationalin 2-hydroxyglutarate (2-HG), a metabolite that participates in tumerogenesis. … [L44256,L44261] It is indicated for the treatment of relapsed or refractory acute myeloid leukemia (AML) in patients with a susceptible IDH1 mutation as determined by an FDA-approved test.