BioMarin Announces EMA Validates MAA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

SAN RAFAEL, Calif., June 25, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for drisapersen for the treatment of Duchenne Muscular Dystrophy amenable to exon 51 skipping. Validation of the MAA confirms that the submission is complete and starts the EMA’s standard review process. Day 120 questions will be received on 22 October 2015, leading to a potential CHMP opinion in the first half of 2016 and a European Commission Decision by the third quarter of 2016.
Drisapersen is an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy (DMD) amenable to single exon skipping. Drisapersen induces the skipping of dystrophin exon 51, potentially providing a therapeutic benefit to DMD patients for whom skipping of exon 51 restores the proper dystrophin reading frame, corresponding to approximately 13% of DMD patients.
“Reaching this important regulatory milestone demonstrates BioMarin’s unwavering commitment to provide a first-in-class therapy for patients who have a specific type of Duchenne muscular dystrophy and have few, if any, treatment options,” said Camilla V. Simpson, Global Head of Regulatory Affairs, Pharmacovigilance. “We are making significant strides on behalf of patients with Duchenne muscular dystrophy. Our hope is to offer a meaningful treatment option around the world.”
DMD is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births. In Europe, it is estimated there are 23,000 boys and young men with Duchenne Muscular Dystrophy, and approximately 3,000 of those would be candidates for drisapersen. In BioMarin’s commercial territories, approximately 85 percent of Duchenne patients are located outside of the United States, including Western Europe, Middle East, Eastern Europe, Latin America and Japan. Western Europe has the largest patient population among those areas, exceeding the United States by around 30 percent. It is estimated that DMD affects approximately 90,000 patients in BioMarin’s commercial territories.
“We are thrilled that BioMarin has reached this point in the EMA review process because it represents a potentially important medical development for boys with Duchenne muscular dystrophy,” said Elizabeth Vroom, Chair of United Parent Projects Muscular Dystrophy (UPPMD). “We are looking forward to the day when boys with Duchenne muscular dystrophy have a number of treatment options that will change the course of this devastating disease.”

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