Sarepta Therapeutics, Inc.’s (SRPT) shares soared over 39% after the company announced it would submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for eteplirsen by year end. Eteplirsen, Sarepta’s lead candidate, is being developed for the treatment of Duchenne muscular dystrophy (DMD).
Sarepta’s decision on the filing of regulatory application for eteplirsen was based on a guidance letter from the FDA. The FDA’s letter and the detailed guidance come as a huge relief for Sarepta. We remind investors that Sarepta’s shares tumbled in November last year after the FDA had stated that the NDA filing of eteplirsen would be premature at that time. The company have had four meetings with the U.S. regulatory body since then. Sarepta now expects the candidate to receive accelerated approval next year.
The U.S. regulatory body, while providing the guidance, asked for additional data to support the efficacy and safety of eteplirsen in DMD treatment. Moreover, the FDA provided a detailed guidance on an open-label, historically controlled confirmatory study of eteplirsen. The FDA also gave initial guidance on a placebo-controlled study of one or more follow-on DMD drug candidates to verify the clinical benefit of eteplirsen.
Following the detailed guidance, Sarepta plans to initiate several studies on eteplirsen in DMD patients later in the year. One of these studies will have predefined efficacy endpoints for ambulatory patients between 7 to 16 years of age who can walk a minimum distance. Two other studies will evaluate the safety and biomarkers in DMD patients less than 7 years of age and in DMD patients whose disease has progressed to a point where they cannot walk a minimum distance or have even become non-ambulant. The FDA also reassured that if the results are positive, the chances of eteplirsen’s NDA acceptance in the U.S. will brighten.
Our Take
We are encouraged by the FDA’s guidance on eteplirsen’s future studies. This will help Sarepta to find the right path for regulatory approval of the candidate. We remind investors that Sarepta Therapeutics currently does not have any approved product in its kitty. The successful development and commercialization of eteplirsen is crucial for the company. We expect investor focus to remain on eteplirsen in the near future.
Sarepta Therapeutics currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks include Amgen Inc. (AMGN), Biogen Idec Inc. (BIIB) and Regeneron Pharmaceuticals, Inc. (REGN). All the stocks carry a Zacks Rank #1 (Strong Buy).
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