BioMarin Announces EMA Validates MAA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

SAN RAFAEL, Calif., June 25, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the European Medicines Agency (EMA) has val­i­dat­ed the Marketing Authorization Application (MAA) for dris­apersen for the treat­ment of Duchenne Muscular Dystrophy amenable to exon 51 skip­ping. Validation of the MAA con­firms that the sub­mis­sion is com­plete and starts the EMA’s stan­dard review process. Day 120 ques­tions will be received on 22 October 2015, lead­ing to a poten­tial CHMP opin­ion in the first half of 2016 and a European Commission Decision by the third quar­ter of 2016.
Drisapersen is an inves­ti­ga­tion­al anti­sense oligonu­cleotide drug can­di­date for the treat­ment of the largest sub­set of Duchenne mus­cu­lar dys­tro­phy (DMD) amenable to sin­gle exon skip­ping. 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.
“Reaching this impor­tant reg­u­la­to­ry mile­stone demon­strates BioMarin’s unwa­ver­ing com­mit­ment to pro­vide a first-in-class ther­a­py for patients who have a spe­cif­ic type of Duchenne mus­cu­lar dys­tro­phy and have few, if any, treat­ment options,” said Camilla V. Simpson, Global Head of Regulatory Affairs, Pharmacovigilance. “We are mak­ing sig­nif­i­cant strides on behalf of patients with Duchenne mus­cu­lar dys­tro­phy. Our hope is to offer a mean­ing­ful treat­ment option around the world.”
DMD is the most com­mon fatal genet­ic dis­or­der diag­nosed in child­hood, affect­ing approx­i­mate­ly 1 in every 3,500 live male births. In Europe, it is esti­mat­ed there are 23,000 boys and young men 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. It is esti­mat­ed that DMD affects approx­i­mate­ly 90,000 patients in BioMarin’s com­mer­cial ter­ri­to­ries.
“We are thrilled that BioMarin has reached this point in the EMA review process because it rep­re­sents a poten­tial­ly impor­tant med­ical devel­op­ment for boys with Duchenne mus­cu­lar dys­tro­phy,” said Elizabeth Vroom, Chair of United Parent Projects Muscular Dystrophy (UPPMD). “We are look­ing for­ward to the day when boys with Duchenne mus­cu­lar dys­tro­phy have a num­ber of treat­ment options that will change the course of this dev­as­tat­ing dis­ease.”

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