BioMarin Completes Rolling NDA Submission to FDA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

SAN RAFAEL, Calif., April 27, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced com­ple­tion of the rolling sub­mis­sion of a New Drug Application (NDA) to the United States Food and Drug Administration (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.
“We believe dris­apersen may offer a mean­ing­ful ben­e­fit to boys liv­ing with DMD whose muta­tions are amenable to exon 51 skip­ping. The total­i­ty of data on dris­apersen con­tains three ran­dom­ized, place­bo-con­trolled, effi­ca­cy tri­als and two long term exten­sion stud­ies, which include some boys treat­ed for approx­i­mate­ly 3.4 years,” said Camilla V. Simpson, Global Head of Regulatory Affairs, Pharmacovigilance. “With this appli­ca­tion, BioMarin con­tin­ues in its long-stand­ing tra­di­tion of devel­op­ing impor­tant ther­a­pies for those who are most in need. The sub­mis­sion of the NDA rep­re­sents a sig­nif­i­cant mile­stone for BioMarin, and we appre­ci­ate the strong, col­lab­o­ra­tive effort of many hard work­ing employ­ees, inves­ti­ga­tors, patients and their fam­i­lies. We look for­ward to work­ing with the U.S. Regulatory Authorities to thor­ough­ly under­stand the data gen­er­at­ed for this het­eroge­nous and crit­i­cal­ly ill patient pop­u­la­tion and hope­ful­ly to bring this treat­ment to patients expe­di­tious­ly.”

Drisapersen has been grant­ed Orphan and Fast Track sta­tus, as well as Breakthrough Therapy des­ig­na­tion by the FDA.

DMD is caused by a muta­tion in the gene that encodes for dys­trophin, a pro­tein that is impor­tant in con­nect­ing the cytoskele­ton of mus­cle fibers to the extra­cel­lu­lar matrix. Its defi­cien­cy in DMD leads to pro­gres­sive mus­cle weak­ness, loss of ambu­la­tion in ear­ly ado­les­cence, and typ­i­cal­ly death due to pul­monary or car­diac insuf­fi­cien­cy in the late twen­ties. Because the Duchenne gene is found on the X-chro­mo­some, it pri­mar­i­ly affects boys; how­ev­er, it occurs across all races and cul­tures. There is cur­rent­ly no approved ther­a­py in the United States for DMD.

“This is a first for the Duchenne com­mu­ni­ty, and we are filled with hope that there could be a treat­ment for Duchenne in the United States,” said Debra Miller, co-founder and CEO of CureDuchenne. “CureDuchenne has been sup­port­ing the devel­op­ment of dris­apersen for more than a decade, and we are delight­ed that BioMarin has reached this impor­tant stage. We salute the researchers who have been work­ing so hard, and we share their deter­mi­na­tion to find a cure for Duchenne.”

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