BioMarin files for EU approval of drisapersen for Duchenne MD

USA-based BioMarin Pharmaceutical (Nasdaq: BMRN) has submitted a Marketing Authorization Application to the European Medicines Agency 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 around one 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 DMD, and about 3,000 of those would be candidates for drisapersen. In BioMarin's commercial territories, around 85% of Duchenne patients are located outside of the USA, including Western Europe, Middle East, Eastern Europe, Latin America and Japan. Western Europe has the largest patient population among those areas, exceeding the USA by around 30%. The company recently submitted a New Drug Application to the US Food and Drug Administration for drisapersen (The Pharma Letter April 28).

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