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Odevixibat
go.drugbank.com/drugs/DB16261ApprovedInvestigationalOdevixibat, or A4250, is an ileal sodium/bile acid cotransporter inhibitor indicated for the treatment of pruritus in patients older than 3 months, with progressive familial intrahepatic cholestasis (PFIC … [A236808,L34793] Odevixibat is the first approved non-surgical treatment option for PFIC.[L34803] Previous therapies for PFIC included a bile acid sequestrant such as [ursodeoxycholic acid].
Capecitabine
go.drugbank.com/drugs/DB01101ApprovedInvestigationalCapecitabine is an orally-administered chemotherapeutic agent used in the treatment of metastatic breast and colorectal cancers. … Capecitabine is a prodrug, that is enzymatically converted to fluorouracil (antimetabolite) in the tumor, where it inhibits DNA synthesis and slows growth of tumor tissue.
Mirdametinib
go.drugbank.com/drugs/DB07101ApprovedInvestigational[L52485] NF1 is an autosomal-dominant genetic condition caused by loss-of-function variants in the _NF1_ tumour suppressor gene,[A265065,A265070] leading to neurofibromin dysfunction and aberrant activation … Mirdametinib is a mitogen-activated protein kinase (MAP2K, MEK, MAPKK) inhibitor. On February 11, 2025, mirdametinib was approved by the FDA for the treatment of neurofibromatosis type 1 (NF1).
Synonyms: N-(((R)-2,3-DIHYDROXYPROPYL)OXY)-3,4-DIFLUORO-2-((2-FLUORO-4-IODOPHENYL)AMINO)BENZAMIDE N-((2R)-2,3-DIHYDROXYPROPOXY)-3,4-DIFLUORO-2-(2-FLUORO-4-IODOANILINO)BENZAMIDE, (-)-N-(((R)-2,3-DIHYDROXYPROPYL)OXY)-3,4-DIFLUORO-2-((2-FLUORO-4-IODOPHENYL)AMINO)BENZAMIDEBaxdrostat
go.drugbank.com/drugs/DB19090ApprovedInvestigationalOn May 18, 2026, baxdrostat was approved by the FDA as a first-in-class aldosterone synthase inhibitor for the treatment of hypertension in combination with other antihypertensive drugs [L56189]. … Baxdrostat is an orally bioavailable, highly selective, first-in-class small-molecule aldosterone synthase inhibitor (ASI) designed to block the production of aldosterone [A275512].
Synonyms: (+)-(r)-n-(4-(1-methyl-2-oxo-1,2,3,4-tetrahydroquinolin-6-yl)-5,6,7,8-tetrahydroisoquinolin-8-yl)propionamide, N-((8r)-5,6,7,8-tetrahydro-4-(1,2,3,4-tetrahydro-1-methyl-2-oxo-6-quinolinyl)-8-isoquinolinyl)propanamideSepiapterin
go.drugbank.com/drugs/DB16326ApprovedInvestigational[L53548,L53593] Sepiapterin was granted marketing authorization by the European Commission in June 2025 and by the US FDA in July 2025 for the treatment of adults and children with phenylketonuria. … Sepiapterin is a small molecule activator of phenylalanine hydroxylase (PAH) used to reduce phenyalanine levels in patients with phenylketonuria.
Etretinate
go.drugbank.com/drugs/DB00926ApprovedWithdrawnEtretinate was taken off the market in Canada in 1996 and America in 1998 due to the risk of birth defects. Etretinate is now used to treat T-cell lymphomas. … Etretinate is a medication used to treat severe psoriasis. It is a synthetic aromatic retinoid.
Levamisole
go.drugbank.com/drugs/DB00848ApprovedInvestigationalVet approvedWithdrawn[A178117] It was manufactured by _Janssen_ and first used in 1969 as an agent to treat worm infestations[A178123] Levamisole was approved by the FDA in 1990 as an adjuvant treatment for colon cancer. … [A178123] Interestingly, levamisole has been found as an adulterant in cocaine and can lead to a variety of adverse effects in individuals using this drug.[A178120]
Products: PVP- LEVAMISOLE POUR-ON SOLUTION 20 %w/vFlotufolastat F-18
go.drugbank.com/drugs/DB17851ApprovedInvestigational, and a high level of accumulation in tumors. … [A259846] In May 2023, the FDA approved the use of flotufolastat F-18 for PET of PSMA-positive lesions in men with prostate cancer with suspected metastasis who are candidates for initial definitive
Rusfertide
go.drugbank.com/drugs/DB17724ApprovedInvestigationalRusfertide is a hepcidin mimetic used to treat erythrocytosis in adults with polycythemia vera. … [L64098] The FDA approved rusfertide on August 28, 2026 for the treatment of erythrocytosis in adults with polycythemia vera, a blood cancer.
Momelotinib
go.drugbank.com/drugs/DB11763ApprovedInvestigationalMomelotinib works to block the JAK-signal transducer and activator of transcription (STAT) signalling pathway, which is aberrant in MF.[A261561, A261566, A261571] … Momelotinib is a Janus Kinase 1 (JAK1) and 2 (JAK2) inhibitor. It is a competitive inhibitor of JAK ATP binding.
Synonyms: N-(CYANOMETHYL)-4-(2-(4-MORPHOLINOANILINO)PYRIMIDIN-4-YL)BENZAMIDE