BioMarin Announces FDA Advisory Committee to Review Drisapersen for Treatment of Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Advisory Committee Meeting Scheduled for November 24, 2015

SAN RAFAEL, Calif., Oct. 15, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (BMRN) today announced that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review the New Drug Application (NDA) for dris­apersen. The FDA is cur­rent­ly review­ing dris­apersen for the treat­ment of patients with Duchenne mus­cu­lar dys­tro­phy amenable to exon 51 skip­ping. The advi­so­ry com­mit­tee will review dris­apersen data includ­ed in a new drug appli­ca­tion (NDA) dur­ing a meet­ing on November 24, 2015.
“Duchenne mus­cu­lar dys­tro­phy is a dead­ly, pro­gres­sive mus­cle dis­or­der with lim­it­ed treat­ment options that affects most­ly boys and young men. We wel­come the nec­es­sary reg­u­la­to­ry review process­es to move treat­ment for this com­mu­ni­ty beyond sup­port­ive care to a ther­a­py that address­es the under­ly­ing cause of the dis­ease,” said Camilla V. Simpson, Global Head of Regulatory Affairs, Pharmacovigilance. “We look for­ward to dis­cussing the New Drug Application for dris­apersen with the advi­so­ry com­mit­tee with the goal of bring­ing this much-need­ed treat­ment one step clos­er to patients who have a spe­cif­ic type of Duchenne mus­cu­lar dys­tro­phy.”
Drisapersen is an inves­ti­ga­tion­al anti­sense oligonu­cleotide drug can­di­date for the treat­ment of the largest sub­set of Duchenne mus­cu­lar dys­tro­phy patients amenable to sin­gle exon skip­ping. In the U.S., it is esti­mat­ed there are approx­i­mate­ly 2,000 patients who might ben­e­fit from treat­ment with dris­apersen.
The Prescription Drug User Fee Act (PDUFA) action date for com­ple­tion of FDA review of the dris­apersen NDA 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. The FDA has also grant­ed dris­apersen Orphan and Fast Track sta­tus, as well as Breakthrough Therapy des­ig­na­tion.

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