Sarepta Therapeutics Inc (NASDAQ:SRPT) said Monday the U.S. Food and Drug Administration asked the biotech company to submit additional data for its planned New Drug Application for the approval of its drug eteplirsen.
Following the announcement, Sarepta’s stock plunged over 30% in premarket trading Monday.
Sarepta Therapeutics got fresh lease of life in April
Sarepta Therapeutics Inc (NASDAQ:SRPT) has suffered a series of setbacks towards its efforts to win FDA approval.
In April, Sarepta disclosed that it had received guidance from the Food and Drug Administration that it could resubmit for regulatory approval of its Duchenne muscular dystrophy (DMD) drug eteplirsen before finishing a larger, confirmatory clinical trial. Mirroring the positive announcement, the biotech company’s shares rose 50% on April 21, 2014.
However, earlier in November, its stock crashed 64% in a single day after the company announced the FDA considered its plans to file for approval of eteplirsen based on existing data premature.
In July, the biotech firm’s shares rose on news regarding an FDA note responding to a petition that was started on the White House website to encourage research on Duchenne muscular dystrophy.
More discussion needed
In its announcement Monday, the company disclosed the FDA states that more discussion was needed to determine what constitutes a complete application for the experimental muscle disorder drug.
DMD is a degenerative disorder that hampers muscle movement and affects one in 3,600 newborn boys, most of whom die by age 30. Eteplirsen is being developed to treat DMD.
In a statement Monday, the biotech company revealed that the FDA guidance also sought results from an independent assessment of dystrophin images and the 168-week clinical data from study 202. Moreover, the guidance also seeks more specific data including a minimum duration of safety in new patients exposed to eteplirsen, a patient-level natural history data to be obtained by Sarepta Therapeutics Inc (NASDAQ:SRPT) from independent academic institutions, and MRI data from a recent study conducted by an independent academic group.
The developer of innovative RNA-based therapeutics indicated that it plans to submit the NDA by mid-year 2015, pending any additional requests from further discussions with the FDA.