On April 25, 2016, the Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) voted that Sarepta Therapeutics, Inc. (Sarepta) has not provided substantial evidence from adequate and well-controlled studies that eteplirsen induces production of dystrophin to a level that is reasonably likely to predict clinical benefit, by a vote of 3-Yes to 7-No, with 3 abstentions.
The Committee also narrowly voted that the clinical results of the single historically-controlled study (Study 201/202) do not provide substantial evidence that eteplirsen is effective for the treatment of Duchenne muscular dystrophy (DMD), by a vote of 6-Yes to 7-No, with no abstentions.
Sarepta had submitted an application for eteplirsen for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping, under new drug application (NDA) 206488.
The PDUFA goal date for the FDA decide on whether to approve the application is May 26, 2016.
See the SAC Tracker report