Sarepta plummets on FDA briefing documents

Sarepta Therapeutics Inc. (NASDAQ:SRPT) fell $17.35 (55%) to $14.28 on Friday after FDA released briefing documents that raised questions about the data package for the company's eteplirsen ( AVI-4658) to treat Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. FDA's Peripheral and Central Nervous System Drugs Advisory Committee is scheduled to meet Jan. 22 to discuss an NDA for eteplirsen, which induces exon 51 skipping on the dystrophin gene. Eteplirsen's PDUFA date is Feb. 26.

Sarepta is making its case for eteplirsen primarily on data from the 12-patient, placebo-controlled Phase IIb Study 201 and its open-label extension, Study 202. An increase in dystrophin-positive muscle fibers was the primary endpoint (see BioCentury, Dec. 7). ...