Sarepta Therapeutics, Inc. SRPT has an important regulatory event coming up next month (Jan 22, 2016) with the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee expected to review the company’s new drug application (NDA) for its lead pipeline candidate, eteplirsen.
Eteplirsen is under priority review in the U.S. for the treatment of patients suffering from Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The FDA is expected to render a final decision on the candidate by Feb 26, 2016.
We remind investors that BioMarin Pharmaceutical Inc. BMRN had announced earlier this month that the FDA has not yet completed its review process for the company’s NDA for the DMD candidate, Kyndrisa. The regulatory agency was expected to announce its verdict by Dec 27, 2015 but pushed off the review date and is now expected to render a decision in early Jan 2016.
The FDA’s advisory panel vote on Kyndrisa, which was held in Nov 2015, was not found to be favorable and they did not find the data compelling enough to support Kyndrisa efficacy. Out of the 17 panel members, 15 were of the opinion that the lack of statistical significance in BioMarin's late-stage study on Kyndrisa weakened the findings from the two earlier studies.
Considering the feedback that BioMarin received from the advisory panel regarding Kyndrisa, the advisory panel’s review of Sarepta’s eteplirsen is crucial. With no approved product in Sarepta’s portfolio and eteplirsen being the lead pipeline candidate in its pipeline, the company is entirely dependent on eteplirsen for growth.
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. It’s to be seen who gets a head start in this race and ends up capturing a significant market share for this indication.
We expect investor focus to remain on the outcome of the upcoming advisory panel meeting and approval status of eteplirsen and Kyndrisa.
Currently, both BioMarin and Sarepta are Zacks Rank #3 (Hold) stocks. A couple of better-ranked stocks in the health care sector are Achillion Pharmaceuticals, Inc. ACHN and Anika Therapeutics Inc. ANIK, both carrying a Zacks Rank #1 (Strong Buy).
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