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This FDA Letter About Sarepta and Accelerated Approval for Eteplirsen Will Blow Your Mind

Sarepta Therapeutics  (SRPT) shares cratered in November after the FDA told the company not to seek accelerated approval for its Duchenne muscular dystrophy drug eteplirsen. 

But what if FDA is changing its mind? 

FDA official Catherine Chew is sending a very interesting letter to people who write the agency in support of eteplirsen's early approval. 

I've reprinted the full text of the letter below, but check out this paragraph:

We understand that you feel that eteplirsen is highly effective, and may be confused by what you have read or heard about FDA's actions on eteplirsen. Unfortunately, the information reported in the press or discussed in blogs does not necessarily reflect FDA's position. 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. [Emphasis added.]

Um... wow! 

Now, let's not get too excited. There has been no indication from Sarepta about a change in regulatory strategy, meaning the company is still focused on finalizing the design of the required eteplirsen phase III study. An agreement with FDA on  that front is expected later this quarter. 

However, Chew's letter, suggests some wiggle room for the possibility that FDA would... might... possibly... be willing to review eteplirsen based on the existing phase II data. 

In fact, the same could be said for Prosensa  (RNA) and GlaxoSmithKline's  (GSK) drisapersen, if the companies decide to submit data from the failed phase III study. 

Sarepta shares are down 10% to $18.33 today because Citibank analyst Yaron Werber downgraded the stock to a sell. He doesn't believe eteplirsen has a chance in hell of receiving an early FDA review. In fact, he doesn't expect eteplirsen to be approved before 2017. 

Werber's view is close to consensus. But then there's this FDA letter making the rounds, so maybe Sarepta's door isn't shut entirely. 

The full text of Chew's letter is below:

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, and may be confused by what you have read or heard about FDA's actions on eteplirsen. Unfortunately, the information reported in the press or discussed in blogs does not necessarily reflect FDA's position. 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's analyses of eteplirsen and other drugs for the treatment of Duchenne muscular dystrophy have been 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, and this makes it difficult for you to understand FDA's evolving position on these drugs. We know this is very frustrating, and we think this may have caused some of the misunderstanding here.

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 understand 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.

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

Sincerely,

Catherine Chew, PharmD
Acting Director, Division of Drug Information 
Center for Drug Evaluation and Research
Food and Drug Administration


-- Reported by Adam Feuerstein in Boston.  

Follow Adam Feuerstein on Twitter.

Stock quotes in this article: SRPT, RNA, GSK 
Adam Feuerstein writes regularly for TheStreet. In keeping with company editorial policy, he doesn't own or short individual stocks, although he owns stock in TheStreet. He also doesn't invest in hedge funds or other private investment partnerships. Feuerstein appreciates your feedback; click here to send him an email.

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