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Palovarotene
go.drugbank.com/drugs/DB05467ApprovedIt first garnered interest as a potential treatment for emphysema but was eventually recognized as a potential novel therapy for patients with FOP. … [L39990,L40020] It has been granted rare pediatric disease and breakthrough therapy designations from the US FDA, although a previously submitted NDA was withdrawn in August 2021 pending the resubmission
Oxycodegol
go.drugbank.com/drugs/DB14146InvestigationalLoxicodegol was developed by Nektar Therapeutics as an opioid analgesic with low abuse potential for the treatment of chronic pain [L3263].
Fexinidazole
go.drugbank.com/drugs/DB12265Approved[A237555, L35940] It is currently marketed by Sanofi-Aventis.[L35940] … all-oral treatment for first and second stage HAT caused by _T. brucei gambiense_.
Categories: MATE 2-K InhibitorsOnasemnogene abeparvovec
go.drugbank.com/drugs/DB15528ApprovedInvestigational[Nusinersen] is another gene therapy that is currently approved by the FDA for the treatment of SMA in pediatric and adult patients. … Developed by AveXis, a Novartis company,[A187253] onasemnogene abeparvovec is commonly marketed as Zolgensma®, which is available as a single-dose intravenous infusion.
Nomifensine
go.drugbank.com/drugs/DB04821ApprovedWithdrawnAlso withdrawn from the Canadian and UK markets.
Abametapir
go.drugbank.com/drugs/DB11932ApprovedThe necessity for follow-up treatment may lead to challenges with patient adherence, and resistance to agents like permethrin and [pyrethrins]/[piperonyl butoxide] may be significant in some areas.
AV608
go.drugbank.com/drugs/DB05072InvestigationalAV608 is a NK-1 antagonist. It is developed for the treatment of Social anxiety disorder (SAD), irritable bowel syndrome (IBS) and overactive bladder (OAB).
Pibrentasvir
go.drugbank.com/drugs/DB13878ApprovedInvestigationalMavyret is also indicated for HCV genotype 1-infected patients who have been previously treated with regimens either containing an NS5A inhibitor or an NS3/4A protease inhibitor, but not both [L940]. … FDA approved an expanded indication for Mavyret, making it the first and only direct-acting antiviral therapy approved for the treatment of both acute and chronic HCV in patients aged three years and older
Chymotrypsin
go.drugbank.com/drugs/DB09375ApprovedInvestigationalVet approvedWithdrawnChymotrypsin is the most abundant pancreatic proteases that represent up to 10-20% of the total protein synthesized by the exocrine pancreas [A32637].
Iptacopan
go.drugbank.com/drugs/DB16200ApprovedInvestigational[A262581] On December 6th, 2023, Iptacopan under the brand name Fabhalta was approved by the FDA for the treatment of adults with PNH. … Iptacopan is a small-molecule factor B inhibitor previously investigated as a potential treatment for the rare blood disease paroxysmal nocturnal hemoglobinuria (PNH) by inhibiting the complement factor