Application Nr Approved Date Route Status External Links
NDA206488 None Intravenous None Google , Wikipedia , PubMed , DailyMed , Raw OpenFDA , ECHA , ToxNet , JECFA , FAO , 21 CFR

Indications and Purposes

1 Indications And Usage Exondys 51 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 51 Skipping. This Indication Is Approved Under Accelerated Approval Based On An Increase In Dystrophin In Skeletal Muscle Observed In Some Patients Treated With Exondys 51 [See Clinical Studies ( 14 )] . Continued Approval For This Indication May Be Contingent Upon Verification Of A Clinical Benefit In Confirmatory Trials. Exondys 51 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 51 Skipping. This Indication Is Approved Under Accelerated Approval Based On An Increase In Dystrophin In Skeletal Muscle Observed In Some Patients Treated With Exondys 51 [See Clinical Studies ( 14 )]. 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. Eteplirsen

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