BioMarin's NDA for DMD Drug Drisapersen Accepted by FDA - Analyst Blog
BioMarin Pharmaceutical Inc. BMRN announced that its New Drug Application for drisapersen has been accepted for review by the FDA for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
The FDA granted a priority review status to drisapersen – a final response from the agency should be out by Dec 27, 2015.
Meanwhile, the FDA informed the company that it plans to hold an advisory committee meeting to discuss the application. The date of the meeting however is not yet decided.
Drisapersen became a part of BioMarin’s pipeline following its Jan 2015 acquisition of Prosensa. It has orphan drug status in the U.S. as well as fast track status and Breakthrough Therapy designation.
The news about the FDA’s acceptance of the NDA comes a few days after the company announced that its Marketing Authorisation Application has been validated by the European Medicines Agency (EMA). The validation marks the initiation of the EMA's standard review process. The company is expecting to receive Day 120 questions on Oct 22, 2015, followed by an opinion from the Committee for Medicinal Products for Human Use in the first half of 2016. A final decision from the European Commission is expected by the third quarter of 2016.
Per the company, DMD is estimated to affect about 1 in every 3,500 live male births. Drisapersen induces the skipping of dystrophin exon 51, corresponding to an approximate 13% of all DMD patients. The company estimates that there are approximately 20,000 new cases diagnosed across the world every year.
We note that companies like Sarepta Therapeutics, Inc. SRPT are also working on bringing their DMD treatments to market. Sarepta finished submitting its regulatory application for its own DMD candidate, eteplirsen, in the U.S. on Jun 26, 2015. Sarepta is also seeking priority review for eteplirsen, which has orphan drug status as well as fast track status in the U.S.
BioMarin currently carries a Zacks Rank #3 (Hold). Valeant Pharmaceuticals International, Inc. VRX and Gilead Sciences Inc. GILD are better-ranked stocks in the health care sector, each carrying a Zacks Rank #1 (Strong Buy).
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