About eteplirsen FDA commissioner responds to mothers’ plea

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.

Read more reports 'Straight from Sharman'

Read more reports ‘Straight from Sharman’


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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