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We the people ask the federal government to Take or explain a position on an issue or policy:

Urge the FDA to Say YES to Accelerated Approval for safe, effective therapies for children with Duchenne.

Created by A.C. on February 27, 2014

We urge the Food and Drug Administration (FDA) to use the Accelerated Approval pathway for approval and access to safe, effective therapies for Duchenne Muscular Dystrophy - the leading genetic killer of children that impacts 1 out of every 3,500 boys born in the United States. It's time for the FDA to Say Yes and make this the first generation of Duchenne survivors.

Health Care

Response to Petition

Drug Approval Pathways and Duchenne Muscular Dystrophy

By Janet Woodcock

Thank you for your petition on Duchenne muscular dystrophy.

We know that Duchenne muscular dystrophy is a serious and life-threatening disease, severely impacting patients and their families, for which there are no FDA-approved therapies. We are willing to explore the use of all potential pathways for the approval of drugs for Duchenne muscular dystrophy, including accelerated approval, as appropriate.

We share your sense of urgency to make safe and effective drugs available for patients with Duchenne muscular dystrophy as soon as possible. That's why we're actively engaged with a number of drug companies focused on developing new drugs for Duchenne muscular dystrophy, including Sarepta Therapeutics, the company developing eteplirsen, an investigational new drug for Duchenne muscular dystrophy.

Our ongoing analyses of eteplirsen and other drugs for the treatment of Duchenne muscular dystrophy are based on rigorous assessments by a large multi-disciplinary team of scientists. Although our assessments are rigorous and extensive, we recognize the urgency of the needs of patients with Duchenne muscular dystrophy, and we carry out our analyses expeditiously.

Sarepta Therapeutics has publicly announced its intention to file a New Drug Application for eteplirsen by the end of 2014 as well as plans to initiate several additional clinical studies with eteplirsen later this year. As mentioned above, we are willing to explore the use of all potential pathways for the approval of drugs for Duchenne muscular dystrophy (including accelerated approval) as appropriate.

You may be aware that FDA recently participated in a policy forum with the Duchenne community, including patients, parents, experts, and other stakeholders. We will continue to work with the community to advance the development of safe and effective treatments for Duchenne muscular dystrophy.

Thank you again for taking the time to write to us.

Janet Woodcock is Director, FDA Center for Drug Evaluation and Research

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