FOX follow up: FDA commissioner responds to mothers' plea


BOSTON ( -- FDA Commissioner Margaret Hamburg responded Friday to the pleas of two local mothers trying desperately to get approval for a drug to treat their children who have a terminal illness.

Fox25 Political Reporter Sharman Sacchetti asked Hamburg about Jenn McNary and Christine McSherry, two mothers who are pleading for the FDA to act on a drug approval for their sons with terminal illnesses.

In an emotional plea on FOX 25, the mothers' asked the FDA to approve a drug called eteplirsen, made by the Cambridge-based company, Sarepta Therapeutics. They say it would help their children with Duchenne Muscular Dystrophy.

Thursday one of the moms told FOX 25 that her one son on the drug is doing well, the other, who is not taking it, is not doing as well. The illness is terminal. When we asked the FDA commissioner what the holdup was, she said the company needs to submit an application. It's a complex process, and the mothers' say the FDA needs to first provide guidance to that company saying it has enough data to file an application.

FOX 25 confronted the commissioner at the MassBio annual meeting in Cambridge, where she appeared alongside Senator Elizabeth Warren. Sarepta Therapeutics of Cambridge, said they're working with the FDA on the design of a larger study of the drug, that would include more patients at a number of clinical sites.

They plan to begin the study in the second quarter of the year, with dosing in patients to begin in the months following.

Fox25 will continue following this story and will bring you any updates.

Below is the text of a letter that was sent to those who have contacted the FDA about eteplirsen:

Thank you for your message to the Food and Drug Administration (FDA) about eteplirsen, a new drug under investigation for the treatment of Duchenne muscular dystrophy. We recognize the huge unmet medical need in Duchenne muscular dystrophy, the devastating nature of the disease for patients and their families, and the great urgency to make new treatments available.

We understand that you feel that eteplirsen is highly effective. FDA's ongoing analyses of eteplirsen and other drugs for the treatment of Duchenne muscular dystrophy are based on thorough and extensive assessments and discussion of all available data and information by a large multi-disciplinary team of FDA scientists. Many assessments are ongoing. Some of this information, in particular, information about the dystrophin biomarker, the natural progression of the disease, and the results from clinical studies, are not available to the public. We are prevented by legal regulations from divulging this information, or our assessment of this information, making it hard to understand FDA's evolving position on these drugs. Please know that FDA has reached no conclusions about the possibility of using accelerated approval for any new drug for the treatment of Duchenne muscular dystrophy, and for eteplirsen in particular.

FDA is fully committed to make safe and effective drugs available for patients with Duchenne muscular dystrophy as soon as possible, and is actively engaged with all drug companies developing new drugs for Duchenne muscular dystrophy, including Sarepta Therapeutics, the company developing eteplirsen.

As you may know, we recently participated in a policy forum with the Duchenne community, including patients, parents, experts, and other stakeholders. Please know that we hear your frustration, and fully understand the dire urgency of the situation. We will continue to work relentlessly with the community to make safe and effective treatments for Duchenne muscular dystrophy available to patients.

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