BioMarin Pharmaceutical Inc. BMRN announced that its Marketing Authorisation Application (MAA) for pipeline candidate, drisapersen, has been validated by the European Medicines Agency (EMA). The company is looking to get drisapersen approved for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
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 and a final decision from the European Commission by the third quarter of 2016.
We note that the company has already submitted a New Drug Application in Apr 2015 for drisapersen. Drisapersen became a part of BioMarin’s pipeline following its Jan 2015 acquisition of Prosensa. It has orphan drug status as well as fast track status and has been granted Breakthrough Therapy designation by the FDA.
According to information provided by the company in its press release, DMD is estimated to affect about 1 in every 3,500 live male births. In Europe, about 23,000 boys are estimated to suffer from DMD – the company believes that approximately 3,000 of those would be candidates for drisapersen.
The company estimates that across its commercial territories, approximately 90,000 patients are affected by DMD with 85% of them being located in ex-U.S. territories including Western Europe, the Middle East, Eastern Europe, Latin America and Japan. The largest patient population is in Western Europe exceeding the U.S. by about 30%.
We note that companies like Sarepta Therapeutics SRPT are also working on bringing DMD treatments to market. Sarepta is looking to finish submitting the regulatory application for its DMD candidate, eteplirsen, in the U.S. by mid- 2015.
Meanwhile, we remain encouraged by BioMarin’s progress with its pipeline. BioMarin’s shares achieved a 52-week high earlier this week reflecting the impact of the positive results from a phase II proof-of-concept and dose finding study on BMN 111(dwarfism).
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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