Sarepta Therapeutics, Inc.
) 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).
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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
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.
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
Biogen Idec Inc.
Regeneron Pharmaceuticals, Inc.
). All the stocks carry a Zacks Rank #1 (Strong Buy).