A note from Debra

BioMarin Update to the Duchenne Community-November 25, 2015

Posted on November 26, 2015 12:52 am


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 patientadvocacy@bmrn.com or:

North America, Latin America: Celia Economides: celia.economides@bmrn.com

Europe, Middle East, Africa, Asia Pacific: Paul Humphrey: paul.humphrey@bmrn.com

For individual patients please, contact BioMarin Medical Affairs:

Europe, Middle East, Africa: medinfoeu@bmrn.com Latin America: medinfola@bmrn.com

North America: medinfo@bmrn.com Asia Pacific: medinfoasia@bmrn.com


Posted on November 25, 201512:05 am

CureDuchenne, the California-based nonprofit organization dedicated to finding cures for Duchenne muscular dystrophy, released the following statement from its co-founder and CEO, Debra Miller, following the meeting of the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA). The FDA will consider the committee’s discussions, and has set a Read more »

FDA Advisory Committee Meeting

Posted on November 20, 20154:50 pm

  We are looking forward to the U.S. Food & Drug Administration (FDA) Advisory Committee meeting on  Tuesday, November 24. This will be an important day for the Duchenne community. The committee members selected by the FDA are set to review clinical data for Drisapersen and hear testimony from patients, doctors and caregivers, the individuals Read more »