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Prademagene zamikeracel
go.drugbank.com/drugs/DB17895ApprovedInvestigational[L53013] It was granted an Orphan Drug designation by the EMA.[A273833] … [L53008] Prademagene zamikeracel was first approved by the FDA on April 29, 2025, for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a skin disorder associated with excessive blistering
Temafloxacin
go.drugbank.com/drugs/DB01405ApprovedWithdrawnIt was first approved for use in the U.S. market in 1992, but was withdrawn shortly due to the reports of serious adverse reactions, such as allergic reactions and hemolyric anemia, resulting in three
Lumacaftor
go.drugbank.com/drugs/DB09280ApprovedInvestigational[A20343] Improvements in lung function (ppFEV1) were found to be statistically significant, but minimal, with only a 2.6-3.0% change from baseline with more than 70% of patients failing to achieve an absolute … [A18395] The next most common mutation, G551D, affecting 4-5% of CF patients worldwide, is characterized as a missense mutation, whereby there is sufficient amount of protein at the cell surface, but opening
Pentastarch
go.drugbank.com/drugs/DB09111ApprovedWithdrawnIt is sold under the name Pentaspan by Bristol-Myers Squibb and is used for fluid resuscitation. … Pentastarch is characterized by presenting five hydroxyethyl groups, which signifies an approximate 50% hydroxyethylation.
Albiglutide
go.drugbank.com/drugs/DB09043ApprovedInvestigationalWithdrawnAlbiglutide was approved on April 15, 2014 by the FDA. … It is marketed under the brands Eperzan and Tanzeum by GSK (GlaxoSmithKline). It is a dipeptidyl peptidase-4-resistant glucagon-like peptide-1 dimer fused to human albumin.
Elafibranor
go.drugbank.com/drugs/DB05187ApprovedInvestigational[L50773] The drug was also approved by the EMA on September 23, 2024.[L51878] … [L50768] On June 10, 2024, elafibranor was granted accelerated approval by the FDA for the treatment of primary biliary cholangitis (PBC).
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). Odevixibat is the first approv...
Betula pendula tar oil
go.drugbank.com/drugs/DB11361ApprovedNutraceuticalThis oil is usually obtained by using both hydrodistillation and micro-distillation[A33154] and it is conformed mainly by [DB11143] and [DB11359]. … [L1113] By the FDA, it is considered under the substances approved to be added to the food.
Afamelanotide
go.drugbank.com/drugs/DB04931Approved[L9086] It differs structurally from its endogenous counterpart by only two amino acids - these structural differences improve biological efficacy by imparting a greater affinity for its target and a longer
Glutathione disulfide
go.drugbank.com/drugs/DB03310ApprovedA GLUTATHIONE dimer formed by a disulfide bond between the cysteine sulfhydryl side chains during the course of being oxidized.