BioMarin Update to the Duchenne Community-November 25, 2015


BioMarin expresses its heartfelt appreciation to the Duchenne patient organizations, families, boys and young men living with Duchenne muscular dystrophy (Duchenne) who voiced their perspectives at the November 24th Food and Drug Administration (FDA) Advisory Committee meeting to consider our New Drug Application (NDA) for drisapersen for the treatment of Duchenne amenable to exon 51 skipping.

This is an enormously challenging disorder to study, and we appreciated the opportunity to present to the Committee data from the 326 boys and young men who participated in the drisapersen clinical trials in support of review and approval of our NDA. The Committee’s discussion is one step in the overall journey of the drisapersen development program, and regulatory review and approval of a potential treatment option for those living with Duchenne. Although the Committee discussed the strengths and weaknesses of the data, it was not asked to vote on a recommendation for approval. The Committee’s feedback will be considered by the FDA in its review of the NDA for drisapersen. The FDA is not bound by the Committee’s guidance, but takes its advice into consideration when reviewing investigational medicines.

BioMarin’s next step is to continue working with the FDA as they complete their regulatory review. The FDA has set a target action date of December 27, 2015 for a decision regarding drisapersen’s review.

BioMarin Patient Advocacy

For patient and family group leaders, please contact BioMarin Patient Advocacy at or:

North America, Latin America: Celia Economides:

Europe, Middle East, Africa, Asia Pacific: Paul Humphrey:

For individual patients please, contact BioMarin Medical Affairs:

Europe, Middle East, Africa: Latin America:

North America: Asia Pacific:

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