PPMD Sends Letter to FDA Regarding Duchenne Therapies

Today, PPMD sent a letter to the FDA requesting updates on various regulatory actions of tremendous importance to the Duchenne community. In our letter, we are asking the FDA to talk to our community, tell us what is going on, and confirm that they are taking into account the perspectives and experiences of the hundreds of families that have shared their history with each of these potential therapies.

The clock continues to tick and time passes – time our community doesn’t have.

Read PPMD's Letter to the FDA:

August 11, 2016

Janet Woodcock, MD
Director, CDER
U.S. Food & Drug Administration
10903 New Hampshire Avenue, Room 6133
Silver Spring, MD 20993

Dear Dr. Woodcock:

On behalf of Parent Project Muscular Dystrophy (PPMD), I am writing to request that the FDA take any steps possible to provide an update and clearly communicate the status of various regulatory actions of tremendous importance to the Duchenne community.

As you know, our families are experiencing an unprecedented series of challenges related to potentially conflicting information about the Duchenne therapy development pipeline. We appreciate everything that you and your colleagues have done to engage our community in this process. Nevertheless, several critical issues are unresolved at this time, with potentially dire consequences for our community.

Specifically, we need your assistance to bring greater clarity and consistency to a process that at times appears confusing to many. We recognize that each candidate therapy is reviewed on its own merits and respect that there is a commitment to confidentiality. At the same time, families living with Duchenne face desperate unmet medical needs. We cannot sit back and assume that these regulatory decisions have fully taken parent and patient considerations into account without better information and more detailed explanations.

Our concerns revolve around a cascade of unfavorable and seemingly contradictory decisions over the last several months. First, we experienced the failure of Biomarin’s drisapersen to gain market authorization without the opportunity to have a formal benefit-risk analysis within the review documents by the agency. This was followed closely by the protracted and still ongoing technical scrutiny of Sarepta’s eteplirsen in the wake of an unprecedented Advisory Committee experience for our community. We have also witnessed the refusal to file decision against PTC’s ataluren which would deny that potential therapy its opportunity for a completed review, and finally the denial of an accelerated approval pathway for Santhera’s idebenone, which met its primary endpoint in their clinical trial. In aggregate, these actions have left the Duchenne community greatly concerned about the consistency and transparency of the FDA approval process.

Again, we appreciate the fact that the FDA is making unprecedented efforts to consider the patient perspective in conducting these life-changing decisions. Our request to you now is to move even more completely in this direction by fully explaining these reviews with the insight and authority that only the FDA can bring to our families.

Thank you for considering this request. We are anxiously awaiting your reply and stand ready to provide any assistance that would be useful.


Pat Furlong

cc: Eric Bastings, MD
Billy Dunn, MD
Robert Temple, MD
Ellis Unger, MD 

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