Application Nr Approved Date Route Status External Links
NDA211970 None Intravenous None Google , Wikipedia , PubMed , DailyMed , Raw OpenFDA , ECHA , ToxNet , JECFA , FAO , 21 CFR

Indications and Purposes

1 Indications And Usage Vyondys 53 Is Indicated For The Treatment Of Duchenne Muscular Dystrophy (Dmd) In Patients Who Have A Confirmed Mutation Of The Dmd Gene That Is Amenable To Exon 53 Skipping. This Indication Is Approved Under Accelerated Approval Based On An Increase In Dystrophin Production In Skeletal Muscle Observed In Patients Treated With Vyondys 53 [See Clinical Studies ( 14 )] . Continued Approval For This Indication May Be Contingent Upon Verification Of A Clinical Benefit In Confirmatory Trials. Vyondys 53 Is An Antisense Oligonucleotide Indicated For The Treatment Of Duchenne Muscular Dystrophy (Dmd) In Patients Who Have A Confirmed Mutation Of The Dmd Gene That Is Amenable To Exon 53 Skipping. This Indication Is Approved Under Accelerated Approval Based On An Increase In Dystrophin Production In Skeletal Muscle Observed In Patients Treated With Vyondys 53. Continued Approval For This Indication May Be Contingent Upon Verification Of A Clinical Benefit In Confirmatory Trials. ( 1 )

All Formulated Excipients (0 Total)

None

Active Ingredients ( 1 Total)

Name Structure ZINC ID(s)
1. Golodirsen

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