White House Petition Response: Drug Approval Pathways and Duchenne Muscular Dystrophy

OFFICIAL FOOD AND DRUG ADMINISTRATION RESPONSE TOUrge the FDA to Say YES to Accelerated Approval for safe, effective therapies for children with Duchenne.

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

Tell us what you think about this petition response and We the People.

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Not wanting to sound ignorant,but what does this mean?

Basically, FDA is asking Sarepta Therapeutics to file a New Drug Application with more data but FDA will do whatever they think is appropriate.

I find this letter to be a typical response by a 30 year veteran FDA bureaucrat. 

As we see with the Ebola outbreak FDA can do whenever they want. Fast tracking a treatment, skipping the entire Clinical trial process. 

Eteplirsen has no side effects and has shown to be effective for 144 weeks. With all this evidence where is the DMD drug. 

Hi Jason.Thanks for clearing that up for me,it is one document that left my mind spinning.And yes,from FDAs response on Ebola,i guess one may be forgiven for thinking that to FDA,DMD is not dangerous enough to the general public to guarantee a rapid a response as we are seeing from them with Ebola.Agreed,they do what they want. 

Apples and oranges....

The FDA has provided our community extraordinary access to the process, and clear guidance to Sarepta.  

DMD may not be as an efficient killer as Ebola but DMD patients have lost the ability to walk, lift there arms, and some have died in the 9 months since the FDA Considers NDA Filing Premature. Patients organizations with tremendous political and scientific pressure have made the FDA reconsider.

The Clinical trial process is a good thing but what is the point of Orphan Drug Status and Fast tracking if the FDA won't use it.  Eteplirsen a safe and apparently working drug based on 144 weeks 6MWT and Pulmonary Function Test data.

There is nothing stopping the FDA from approving this drug and still doing a phase 3 trial.

The FDA hasn't done anything that they haven't done before. What you call "clear guidance" looks like political cover if they don't approve the drug.  

I agree with Jason. Words are cheap.

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