Rolling FDA submission for drisapersen for Duchenne muscular dystrophy is completed.-BioMarin

BioMarin Pharmaceutical Inc. announced completion of the rolling submission of a New Drug Application (NDA) to the FDA for drisapersen, an investigational exon-skipping drug candidate for the treatment of the largest genetically defined subset of 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 approximately 13% of DMD patients. The company intends to also submit an application for registration in the European Union in summer 2015.

Comment:Competitor is eteplirsen an exon skipping drug from Sarepta Therapeutics which has positive results. Eteplirsen, has, so far, shown better improvements in six-minute walk test scores than drisapersen for boys with DMD. Sarepta, after discussions with the FDA, plans to file an NDA in mid- 2015.