October 27, 2014
By Riley McDermid, BioSpace.com Breaking News Staff
Sarepta Therapeutics, Inc. is once again roiling capital markets, after the biotech said Monday that it will be delaying its new muscular dystrophy drug as it attempts to provide the U.S. Food and Drug Administration, pushing its share price down more than 30 percent in early morning trading.
This came after a year on which Sarepta has seen its stock skyrocket on news that in April that the FDA might let it leapfrog larger later-stage studies. That status has now not been granted, apparently.
Sarepta said the new FDA request came out of a planned New Drug Application (NDA) submission for the approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD).
After a September NDA meeting, the FDA is now asking Sarepta for the results from an independent assessment of dystrophin images and the 168-week clinical data from study 202.
The regulator is also asking for specific data detailing the minimum duration of safety in new patients exposed to eteplirsen, patient-level natural history data to be obtained by Sarepta from independent academic institutions, and MRI data from a recent study conducted by an independent academic group.
The FDA indicated that further discussion with Sarepta “will be necessary to determine what would constitute a complete NDA.” Based on these requests, Sarepta plans to submit an NDA by mid-year 2015, pending any additional requests from further discussions with the FDA.
Sarepta executives said Monday they remain committed to satisfying the FDA’s updated requests.
“We believe all of the data requests and additional FDA discussions that have currently been outlined can be completed in time for an NDA submission by mid-year 2015,” said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics, in a statement. “Obtaining an FDA approval of eteplirsen for the DMD patients who may benefit from the drug continues to be our highest priority.”
