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Sarepta's Long-Term Phase IIb Data on Eteplirsen Published

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Sarepta Therapeutics, Inc.SRPT announced that results from a long-term open-label extension phase IIb study on its lead pipeline candidate, eteplirsen, were published online in the Annals of Neurology . Eteplirsen is currently under FDA review for the treatment of patients suffering from Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The company's shares were up 5.2% on the news.

Results showed that patients when treated with eteplirsen demonstrated a statistically significant 151 meter difference in the 6-minute walk test compared to the external cohort at 36 months. Patients on eteplirsen witnessed loss in ambulation at a lower rate (16.7%) compared to natural history control patients (46.2%). Patients experienced a slower rate of disease progression when compared to untreated matched historical controls over a treatment period of three years. On the safety front, eteplirsen continued to be well tolerated.

We remind investors that a response from the FDA regarding eteplirsen's approval status is expected by Feb 26, 2016. But before that, eteplirsen will most likely be reviewed by the FDA's Peripheral and Central Nervous System Drugs Advisory Committee, tentatively on Jan 22, 2016.

In addition, Sarepta is looking to seek approval for eteplirsen in the EU next year pending discussions with the European Medicines Agency scheduled before year end.

DMD, a devastating and debilitating disease, is estimated to affect nearly 1 in every 3,500 boys born across the world. There is significant unmet need for DMD treatments. However, Sarepta is not the only company working to bring a treatment for DMD to the market. BioMarin Pharmaceutical Inc. BMRN is also seeking U.S. and EU approval for its DMD candidate, Kyndrisa (drisapersen).

Kyndrisa is scheduled to be reviewed by the FDA's advisory panel next week. BioMarin could have a two-month head start if approval comes by Dec 27, 2015. Kyndrisa is also under review in the EU, where a decision could be out in the second half of 2016.

We expect investor focus to remain on the outcome of the upcoming advisory panel meetings.

Sarepta is a Zacks Rank #3 (Hold) stock. A couple of better-ranked stocks in the health care sector are Actelion Ltd. ALIOF and Baxalta Incorporated BXLT . Both carry a Zacks Rank #1 (Strong Buy).

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.


The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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