Sarepta Receives Negative CHMP Opinion for EXONDYS® (eteplirsen) to Treat Patients with Duchenne Muscular Dystrophy in Europe

Sarepta Therapeutics (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), has adopted a negative opinion for EXONDYS^® (eteplirsen), as expected. EXONDYS is designed to treat patients with Duchenne muscular dystrophy (DMD) amenable to skipping exon 51 of the dystrophin gene.

As quoted in the press release:

As previously reported on Sarepta’s May 5, 2018 earnings call, a negative opinion was anticipated following the oral explanation trend vote. Sarepta will request a re-examination of the opinion, which will result in the assignment of a new rapporteur and co-rapporteur. The Company will also request a Scientific Advisory Group (SAG) on DMD be called so that neuromuscular specialists, experienced with working with treatments for these patients, can provide expert guidance and insight into, among other things, the validity of the external controls used and the importance of certain functional endpoints, including for instance, the relevance of meaningful slowing pulmonary decline in patients with this difficult to treat disease. The re-examination process is expected to be completed by year-end 2018.

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