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Mycophenolic acid
go.drugbank.com/drugs/DB01024ApprovedInvestigational[A249180,A249185] Mycophenolic acid is available as enteric-coated tablets of delayed-release, in an effort to improve upper gastrointestinal adverse events by delaying mycophenolic acid release until … [A249175] This regimen can be used in place of the older anti-proliferative [azathioprine] due to its stronger immunosuppressive potency.
Synonyms: (e)-6-(4-Hydroxy-6-methoxy-7-methyl-3-oxo-5-phthalanyl)-4-methyl-4-hexenoic acidBenzthiazide
go.drugbank.com/drugs/DB00562ApprovedThey inhibit Na+/Cl- reabsorption from the distal convoluted tubules in the kidneys. Thiazides also cause loss of potassium and an increase in serum uric acid. … Thiazides cause vasodilation by activating calcium-activated potassium channels (large conductance) in vascular smooth muscles and inhibiting various carbonic anhydrases in vascular tissue.
Synonyms: 6-chloro-1,1-dioxo-3-(phenylmethylsulfanylmethyl)-4H-benzo[e][1,2,4]thiadiazine-7-sulfonamideAtidarsagene 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) geneSaquinavir
go.drugbank.com/drugs/DB01232ApprovedInvestigationalIn 1995 it became the first protease inhibitor approved by the FDA, followed shortly by ritonavir in 1996, and remains in clinical use today due to a relatively benign adverse effect profile as compared … Saquinavir is an HIV-1 protease inhibitor used in combination with [ritonavir] and other antiretrovirals for the treatment of human immunodeficiency virus-1 (HIV-1) infection.
Nicoboxil
go.drugbank.com/drugs/DB12911ApprovedWithdrawnNicoboxil has been investigated for the treatment of Acute Low Back Pain, where it is typically considered an effective and safe therapeutic option. … Such combination topical analgesics are only available for purchase and use (for humans) in some parts of Europe and Asia, like Germany and Australia [F11, F16].
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.
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.
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
Zolbetuximab
go.drugbank.com/drugs/DB15118ApprovedInvestigational[A264723] Zolbetuximab was approved by the FDA in October 2024 for use in patients with HER2 negative, CLDN18.2-positive gastric and GEJ adenocarcinomas.[L51943,L51923] … [L51923] It is specifically targeted against CLDN18.2, a membrane protein normally found in gastric mucosal cells which is overexpressed in 30-50% of gastric and gastroesophageal junction (GEJ) adenocarcinomas