BioMarin submits NDA for drisapersen

US-based BioMarin Pharmaceutical (Nasdaq: BMRN) has completed the rolling submission of a New Drug Application (NDA) to the US Food and Drug Administration for drisapersen to treat Duchenne muscular dystrophy (DMD).

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. 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 around 13% of DMD patients.

Camilla Simpson, global head of regulatory affairs, pharmacovigilance, said: “We believe drisapersen may offer a meaningful benefit to boys living with DMD whose mutations are amenable to exon 51 skipping. The totality of data on drisapersen contains three randomized, placebo-controlled, efficacy trials and two long term extension studies, which include some boys treated for approximately 3.4 years.”