BioMarin Announces FDA Accepts Drisapersen NDA for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

FDA Grants Priority Review Status; FDA PDUFA Date is December 27, 2015

SAN RAFAEL, Calif., June 29, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the U.S. Food and Drug Administration (FDA) has accept­ed for review the sub­mis­sion of a New Drug Application (NDA) for dris­apersen for the treat­ment of Duchenne mus­cu­lar dys­tro­phy (DMD) amenable to exon 51 skip­ping, and the Prescription Drug User Fee Act (PDUFA) goal date for a deci­sion is December 27, 2015. The FDA has grant­ed dris­apersen Priority Review sta­tus, which is des­ig­nat­ed to drugs that offer major advances in treat­ment, or pro­vide a treat­ment where no ade­quate ther­a­py exists.
In the FDA’s fil­ing com­mu­ni­ca­tion, the Agency informed the com­pa­ny that it is cur­rent­ly plan­ning to hold an advi­so­ry com­mit­tee meet­ing to dis­cuss the appli­ca­tion. No date has been set for this meet­ing. Drisapersen pre­vi­ous­ly has been grant­ed Orphan and Fast Track sta­tus, as well as Breakthrough Therapy des­ig­na­tions by the FDA. The U.S. fil­ing is based on three ran­dom­ized place­bo-con­trolled tri­als and two long-term open-label stud­ies of more than 300 patients in which some boys have been treat­ed for more than three years.
“We are ded­i­cat­ed to bring­ing a mean­ing­ful ther­a­py specif­i­cal­ly for patients with a par­tic­u­lar form of Duchenne to patients all over the world. We are thrilled that BioMarin has reached this impor­tant step in the United States, which comes on the heels of the recent val­i­da­tion of our European fil­ing for dris­apersen,” said Camilla V. Simpson, Global Head of Regulatory Affairs, Pharmacovigilance at BioMarin. “Obtaining Priority Review sta­tus is val­i­da­tion of BioMarin’s com­mit­ment to urgent­ly move treat­ment beyond sup­port­ive care and to address the under­ly­ing cause of the dis­ease. We are thank­ful to the boys and their fam­i­lies who par­tic­i­pat­ed in our clin­i­cal tri­als, which have allowed us to achieve this impor­tant mile­stone for Duchenne patients.”
Drisapersen is an inves­ti­ga­tion­al anti­sense oligonu­cleotide drug can­di­date for the treat­ment of the largest sub­set of 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. In the U.S., it is esti­mat­ed there are approx­i­mate­ly 2,000 patients who would be can­di­dates for dris­apersen.
“Since PPMD’s found­ing more than two decades ago, we have been focused on improv­ing the treat­ment, qual­i­ty of life and long-term out­look for boys with Duchenne mus­cu­lar dys­tro­phy,” said Pat Furlong, President and Founder of Parent Project Muscular Dystrophy. “The com­ple­tion of this reg­u­la­to­ry mile­stone brings the com­mu­ni­ty one step clos­er to what could be the first spe­cif­ic drug ther­a­py to treat Duchenne in the United States. We are hope­ful that this is the begin­ning of a new era of many med­ical advance­ments that will change the course of this dev­as­tat­ing dis­ease.”

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