Sarepta Advisory Committee Meeting


As I prepare to travel to Monday’s Advisory Committee meeting I continue to be disappointed by the FDA Peripheral and Central Nervous System Drugs Advisory Committee’s initial review of Sarepta’s Duchenne muscular dystrophy drug eteplirsen.

Despite this news we need to remember The FDA’s initial reaction is just that – an initial response – and there is evidence still to be presented to the advisory committee.

CureDuchenne provided early funding for the development of eteplirsen, and I look forward to speaking in support of this innovative drug – along with other experts, doctors, scientists, families and patients who all recognize its benefits – at the advisory committee meeting on April 25.

There has been a lot of media coverage on the upcoming meeting.  Here is a sampling of media cover post the release of the briefing documents.

The Wall Street Journal

Boston Business Journal


Boston Globe

While we understand the FDA needs to make sure drugs are safe and effective, we also know the consequence of inaction for those with Duchenne. We are hopeful for an accelerated approval for eteplirsen.

I look forward to seeing Duchenne families from around the country on Monday.  The meeting begins at 8 a.m. and the Open Public Hearing is scheduled for 2 p.m.  It will be an important day for the Duchenne community.

If you are unable to attend in person the meeting webcast can be accessed at the following web address:

Travel safely.  I will see you in Maryland.

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