The Jett Foundation Fighting Duchenne Muscular Dystrophy

Letter to FDA Urging New AdComm Date

February 9, 2016

February 8, 2016

Janet Woodcock, Director FDA Center for Drug Evaluation and Research
10001 New Hampshire Avenue, Hillandale Building, 4th Floor
Silver Spring, MD 20993

William Dunn, Director FDA Division of Neurology Products
10903 New Hampshire Avenue, Building 22, Suite 4346
Silver Spring, MD 20993

 

Dear Dr. Woodcock and Dr. Dunn:
This morning, the Duchenne community learned that the FDA has extended the eteplirsen PDUFA date by three months, to May 26, to allow for a full review of the 4-year clinical effectiveness data from the natural history control submitted by Sarepta Therapeutics on January 8.

Three months in the lifecycle of therapy development may be regulatory standard, but we know that you can appreciate that three months in the lifespan of someone with Duchenne is the difference between ambulation and wheelchair use, and for many, life and death.

We are writing today to ask that you select and announce a date within the new PDUFA timeframe as soon as possible so that our community is assured of the agency’s commitment to the public review of eteplirsen before the Peripheral and Central Nervous System (PCNS) Advisory Committee (AdComm) members.

In the aftermath of the agency’s announcement that eteplirsen’s January 22 AdComm was postponed due to weather, the agency indicated to the Duchenne community that the AdComm would be rescheduled within 30 days of the original date. Now, about two weeks from the end of that implied timeframe, the lack of an announcement of a new AdComm date has created many questions and concerns in our community. A transparent, science-based, fair, and open AdComm meeting – which includes the opportunity to lend our perspectives to further the review process during an Open Public Hearing – must be scheduled as soon as possible.

For our community, every day we are forced to wait for a new AdComm date is a loss – a loss of muscle, a loss of ambulation, a loss of independence, and potentially, a loss of life. We have already waited far too long.

We eagerly await your prompt reply in the form of a Federal Register notice announcing a rescheduled date for the eteplirsen Advisory Committee review.

Sincerely,
CDMD at UCLA/Be Reasonable
Duchenne Alliance
Jett Foundation
Parent Project Muscular Dystrophy
The Race to Yes

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