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CHMP opposes approval of Exondys 51 to treat DMD in Europe

The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency (EMA) — is recommending against Exondys 51 (eteplirsen) as a therapy for Duchenne muscular dystrophy (DMD) patients amenable to skipping exon 51 of the dystrophin gene, the treatment’s developer, Sarepta Therapeutics, has announced.

CHMP, in its negative opinion, questioned the evidence provided, particularly in the study’s small size, use of historical data, and failure to compare treated to placebo patients for beyond 24 weeks.

CHMP Opposes Approval Of Exondys 51 To Treat DMD In Europe