FDA Advisory Committees - FDA Center for Drug Evaluation and Research (CDER) - Peripheral and Central Nervous System Drugs Advisory Committee (PCNS)
The Committee voted that Sarepta Therapeutics, Inc. 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.
The Committee was scheduled to discuss a new drug application for eteplirsen injection for intravenous infusion, submitted by Sarepta Therapeutics, Inc., for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. Tarius will monitor the Federal Register for FDA's announcement of a new date for the meeting. See the FDA Announcement
At this meeting, the FDA did not ask the Committee to vote on whether to recommend approval of drisapersen but to vote on whether key aspects of the data strengthen, weaken, or have no effect on the interpretation of the clinical results. The Agency also asked the Committee to discuss the overall strengths and weaknesses of the data for the proposed indication, in a ninth question that was added on the day of the meeting.
Peripheral and Central Nervous System Drugs Advisory Committee (PCNS)
PCNS reviews and evaluates data concerning the safety and effectiveness of marketed and investigational human drug products for use in the treatment of neurologic diseases and makes appropriate recommendations to the Commissioner of Food and Drugs