BioMarin Pharmaceutical Inc. BMRN announced that it has withdrawn a Marketing Authorisation Application (MAA) for Kyndrisa (drisapersen) from the EU, following discussions with the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), which hinted at the issuance of a negative opinion.
The company was seeking an approval for Kyndrisa for the treatment of Duchenne muscular dystrophy (DMD), amenable to exon 51 skipping.
BioMarin said that it will also discontinue the development of the three follow-on products of Kyndrisa – BMN 044, BMN 045, and BMN 053 – which are currently being evaluated in phase II studies for specific forms of DMD. Nevertheless, the company intends to work on the development of a transition plan for the patients who are currently being treated with these candidates.
Meanwhile, the company stated that it will continue to develop next generation oligonucleotides for the treatment of DMD.
Despite the fact that a MAA was withdrawn from the EU, BioMarin continues to expect to achieve break-even or better results in 2017.
We remind investors that in Jan 2016, the FDA issued a Complete Response Letter (CRL) related to the company’s New Drug Application (NDA) for Kyndrisa for the same indication. The agency rejected the application, citing that Kyndrisa had failed to meet the standard of substantial evidence of effectiveness in treating DMD.
DMD, a devastating and debilitating disease, is estimated to affect nearly 1 in every 3,500–5,000 boys born across the world, indicating significant unmet need for treatments in this space.
We note that Sarepta Therapeutics, Inc. SRPT is also looking to get its DMD drug approved. Last week, the company announced that the FDA has extended the Prescription Drug User Fee Act (PDUFA) date for its DMD candidate, eteplirsen, which was scheduled for May 26. The agency reported that it intends to come up with a decision as early as possible.
Currently, BioMarin carries a Zacks Rank #3 (Hold). Bristol-Myers Squibb Company BMY and ANI Pharmaceuticals, Inc. ANIP are a couple of better-ranked stocks in the health care sector, both sporting a Zacks Rank #1 (Strong Buy).
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