BioMarin Submits Drisapersen MAA to EMA for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

SAN RAFAEL, Calif., June 8, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today 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 United States Food and Drug Administration (FDA) for dris­apersen in April 2015.

“The sub­mis­sion of this appli­ca­tion to the EMA rep­re­sents an impor­tant achieve­ment in BioMarin’s efforts to bring a mean­ing­ful ther­a­peu­tic option to patients and fam­i­lies around the world with Duchenne mus­cu­lar dys­tro­phy. We were able to reach this point because of the extra­or­di­nary effort of the employ­ees at BioMarin, the inves­ti­ga­tors for the clin­i­cal tri­als and most impor­tant, the boys and their fam­i­lies who par­tic­i­pat­ed in the clin­i­cal tri­als,” said Camilla V. Simpson, Global Head of Regulatory Affairs, Pharmacovigilance. “BioMarin has a track record of effi­cient­ly devel­op­ing ther­a­pies in rare genet­ic dis­eases, and we are pleased that we have sub­mit­ted this MAA ahead of the expect­ed time­line. We look for­ward to work­ing with the EMA in the com­ing months with the goal of bring­ing this ther­a­py to patients in need.”

“We applaud BioMarin’s com­mit­ment to devel­op a ther­a­py for Duchenne mus­cu­lar dys­tro­phy,” said Elizabeth Vroom, Chair of United Parent Projects Muscular Dystrophy (UPPMD). “The com­mu­ni­ty has been sup­port­ing research and devel­op­ment of treat­ments, and we are pleased that dris­apersen has been sub­mit­ted for EMA review. We are hope­ful that this ther­a­py will lead not only to an approved ther­a­py, but will fur­ther sci­en­tif­ic advances and the devel­op­ment of oth­er treat­ments for boys with Duchenne.”

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