Shares of $SRPT Halved on FDA Denial

We’re getting multiple downgrades this morning after the FDA voted down SRPT’s muscular dystrophy drug, despite massive outpouring of support by trial patients and their families.

Sarepta Therapeutics, Inc. (SRPT), a developer of innovative RNA-based therapeutics, today announced that the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous System Advisory Committee (PCNSC) met to review the new drug application (NDA) for eteplirsen as a treatment for Duchenne muscular dystrophy amenable to exon 51 skipping. The advisory committee voted 6-7 against the finding of substantial evidence from adequate and well controlled studies that show that eteplirsen induces production of dystrophin to a level that is reasonably likely to predict clinical benefit (FDA Question #2). The advisory committee voted 3 – 7, with three abstentions, against finding substantial evidence based on the clinical results of the single historically controlled study (Study 201/202) that eteplirsen is effective for treatment of DMD (FDA Question #7).

“We would like to thank the hundreds of patients and families who participated in the discussion today, underscoring the critical unmet need of people living with Duchenne.” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “We appreciated the opportunity to present our data to the advisory committee panel and will continue to work with FDA as they complete their review of the eteplirsen NDA. Today more than ever, we remain committed to our mission of bringing a treatment to the Duchenne community.”

Shares are getting destroyed in the pre-market.