Sarepta Therapeutics Inc’s (NASDAQ:SRPT) share price plunged as an advisory committee of the US Food and Drug Administration turned down eteplirsen as a treatment for Duchenne muscular dystrophy.
While not binding on the FDA, the US regulator normally takes advisory committee advice into consideration when reviewing New Drug (NDA) and Biologic License Applications (BLA).
In two key questions, the advisory committee voted 6-7 against that eteplirsen induces production of dystrophin to a level that is reasonably likely to predict clinical benefit and by 3–7 that eteplirsen is effective for treatment of DMD.
“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, Sarepta’s interim chief executive.
“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.”
DMD is a rare degenerative neuromuscular disorder that affects young boys mainly and causes severe progressive muscle loss and premature death.
The PCNSC Advisory Committee recommendation was based on a review of results from the Phase IIb clinical program for eteplirsen and other clinical studies.
Shares fell 36% to US$9.62.