FDA Advisory Committee Meeting


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 with first-hand experience with the treatment. The Committee’s charge is to provide a recommendation to the FDA as to whether there is sufficient evidence to recommend approval of Drisapersen for Duchenne muscular dystrophy amendable to exon 51 skipping. The FDA is not bound to follow the advice of its committees, but it will be considered by the FDA as it continues to review the New Drug Application that BioMarin submitted earlier this year.

Today the FDA posted a briefing book intended to provide an overview of the key considerations and topics to be discussed at the meeting. These materials are available to the public and include assessments of Drisapersen from individual reviewers. Although the review was not positive, this document is not the definitive decision and the FDA has until December 27 to render their approval or non-approval.

Drisapersen is potentially the first drug therapy specifically for Duchenne muscular dystrophy, and it is essential and necessary to thoroughly understand and discuss the data because it is the right thing for to do for patients, caregivers and physicians.  Read BioMarin Quarterly Update here.

Given Drisapersen is the first of the potential treatment for Duchenne to be reviewed by the Committee, you should expect to read or hear sentiments and comments from several different points of view. They will try to read the tea leaves and make predictions.  These are all speculations, we will have to wait until Tuesday to see how this unfolds.

The advisory committee meeting will begin at 8.00 am Eastern Standard Time.  It is a public meeting being held at the FDA White Oak Campus in Silver Spring, MD.  Find the agenda here. Oral presentations from the Duchenne community scheduled between 12:30 p.m. – 2:30 p.m. Duchenne parents and patients will share their stories and express their views on how the drug has impacted their lives.

The meeting will also be webcast live.  Find out details of the webcast here.

The Duchenne community is excited to make our voices heard.  We need safe, effective treatments for our sons with Duchenne.  An approved drug will provide hope for  Duchenne families.

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