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Defibrotide
go.drugbank.com/drugs/DB04932ApprovedInvestigationalIt is marketed under the brand names Dasovas (FM), Noravid, and Prociclide in a variety of countries. In the USA it is was approved in March, 2016 as Defitelio. … Defibrotide is the sodium salt of a mixture of single-stranded oligodeoxyribonucleotides derived from porcine mucosal DNA.
Aminolevulinic acid
go.drugbank.com/drugs/DB00855ApprovedInvestigationalA compound produced from succinyl-CoA and glycine as an intermediate in heme synthesis. It is used as a photochemotherapy for actinic keratosis. [PubChem]
Temozolomide
go.drugbank.com/drugs/DB00853ApprovedInvestigationalIt is currently marketed under the trademark TEMODAR® by Merck.[L32033] … [A229853, A229923] Temozomolide as an adjunct to radiotherapy followed by maintenance dosing remains the standard of care for both Glioblastoma and refractory anaplastic astrocytoma.
Tepotinib
go.drugbank.com/drugs/DB15133ApprovedInvestigational[A228058] MET is a desirable target in the treatment of certain solid tumors as it appears to play a critical role, both directly and indirectly, in the growth and proliferation of tumors in which it is … [L31443,L31473] It is the first oral MET-targeted tyrosine kinase inhibitor to allow for once-daily dosing,[L31473] an advantage that may aid in easing the pill burden often associated with chemotherapeutic
Enasidenib
go.drugbank.com/drugs/DB13874ApprovedInvestigationalPatients eligible for this treatment are selected by testing the presence of IDH2 mutations in the blood or bone marrow. … Enasidenib is an orally available treatment for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with specific mutations in the isocitrate dehydrogenase 2 (IDH2)
Osimertinib
go.drugbank.com/drugs/DB09330ApprovedInvestigationalApproximately 10% of patients with NSCLC have a rapid and clinically effective response to EGFR-TKIs due to the presence of specific activating EGFR mutations within the tumour cells. … [A7927] More specifically, deletions around the LREA motif in exon 19 and exon 21 L858R point mutations are correlated with response to therapy.
Givosiran
go.drugbank.com/drugs/DB15066Approved[A187991] Givosiran is the second-ever FDA-approved member of the siRNA drug class (the first being [patisiran]), a new class of drugs promising an important and exciting step forward in the treatment … in which the overproduction of toxic heme intermediates leads to neuro-, nephro-, and gastrotoxicity.
Flecainide
go.drugbank.com/drugs/DB01195ApprovedInvestigationalWithdrawn[A186880] Flecainide’s development began in 1966 and was first synthesized in 1972 as an attempt to generate new anesthetics. … [A186931] It is used to prevent supraventricular and ventricular arrhythmias, as well as paroxysmal atrial fibrillation and flutter.
Sepiapterin
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. … It is a natural precursor of the enzymatic co-factor tetrahydrobiopterin (BH4), which activates PAH and helps reduce blood phenylalanine levels by enhancing the conformational stability of misfolded PAH
Antipyrine
go.drugbank.com/drugs/DB01435ApprovedAntipyrine is often used in testing the effects of other drugs or diseases on drug-metabolizing enzymes in the liver. (From Martindale, The Extra Pharmacopoeia, 30th ed, p29)