BioMarin Pharma submits MAA to EMA for drisapersen for the treatment of Duchenne muscular dystrophy amenable to single exon skipping.

BioMarin Pharmaceutical Inc. announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for drisapersen, an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy (DMD) amenable to single exon skipping. DMD is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births with about 20,000 new cases diagnosed globally each year. In Europe, it is estimated there are 23,000 boys 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.

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. The company recently submitted a New Drug Application (NDA) to the FDA for drisapersen in April 2015.