Piper Jaffray Goes Overweight Sarepta, Expects FDA To Accept Next Key Drug Filing

Sarepta Therapeutics had held a pre-NDA meeting with the FDA in May for its exon 51 skipping drug, eteplirsen, for the treatment of Duchenne Muscular Dystrophy (DMD). Tentoff belives that the company has fulfilled all requests for information from the FDA since then.

A joint AdCom is expected for both eteplirsen and the exon 51 skipping drug developed by BioMarin Pharmaceutical, Inc. BMRN to be tentatively scheduled for the third week of November.

According to the Morgan Stanley report, “Sarepta previously indicated that its data from fourth biopsies, and 192-week data would be submitted to the FDA and expects these data to be made public before the AdCom.”

Eteplirsen had received Rare Pediatric Disease Designation from the FDA last week, and is on a fast-track status. Tentoff believes that this implies that its PDUFA date could be in late February 2016, while drisapersen’s PDUFA date is scheduled for December 27.

The FDA filings for eteplirsen are based on the Phase IIb “201” and open label, extension “202” studies. The data from these trials show that the drug led to “prolonged periods of stability. Importantly, early treatment produced a significant benefit in 6MWT distance compared to patients receiving delayed treatment out to week 168,” Tentoff reported.

Sarepta Therapeutics is also running several ongoing trials on ambulatory and non-ambulatory exon 51 DMD boys, as well as studies on ambulatory exon 53 boys in Europe.