Rare Disease Report
Patients & Caregivers

Duchenne Advisory Committee - Voting Mixed

APRIL 25, 2016
James Radke, PhD
What a day for the rare disease community. Today, the FDA Advisory Committee met to discuss the safety and efficacy of Sarepta’s eteplirsen in Duchenne boys amenable to exon 51 skipping treatment. And it was a very heated discussion.
On one side, the FDA was extremely critical of the data and argued there are too many unknown variables in the data to scientifically conclude that the drug is effective.
On the other side, Sarepta and their secret weapon – the Duchenne patient community – argued that the drug definitely works and to delay the drug’s approval any longer would be an injustice to the community who had already sacrificed so much to get to this point. In total, 52 people – mostly parents and clinicians who care for Duchenne boys – testified during the open public hearing.
In the end, the Advisory Committee was asked to vote on 5 questions with the final question asking the committee if the drug is effective. And for that question, the voting was very mixed with 3 members abstaining due to the fact that the patients' testimonies showed the drug worked but the clinical data presented by Sarepta was not that persuasive. Voting for the 5 questions are listed below.

1. Has the Applicant provided substantial evidence from adequate and well controlled studies that eteplirsen induces production of dystrophin to a level that is reasonably likely to predict clinical benefit?

  Yes  6
  No  7
 Abstain 0

2. Were decisions to administer the 6-minute walk test (vs. conclusions that the patient could no longer walk) sufficiently objective and free of bias and subjective decision-making by patients, their caregivers, and/or health care professionals to allow for a valid comparison between patients in Study 201/202 and an external control group?

  Yes 5
  No 7
  Abstain 1

3. What is the impact of the North Star Ambulatory Assessment results on the persuasiveness of the findings in Study 201/202?

  Stenghten  1
  Weaken 5
  No effect 7

4. What is the impact of the other tests of physical performance (e.g., rise time, 10-meter run/walk) on the persuasiveness of findings in Study 201/202?

  Stenghten  1
  Weaken 2
  No effect 10

5. Do the clinical results of the single historically-controlled study (Study 201/202) provide substantial evidence (i.e., evidence from adequate and well-controlled studies or evidence from a single highly persuasive adequate and well-controlled study that is accompanied by independent findings that substantiate efficacy) that eteplirsen is effective for the treatment of DMD?


 Yes  3
 No  7
 Abstain  3

The FDA has until May 26th to decide on whether to approve the drug or not.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a progressive muscle disorder caused by the lack of functional dystrophin protein. Patients with Duchenne muscular dystrophy lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties.

If it weren't for bad news...

There are an estimated 35,000 patients with Duchenne muscular dystrophy in the United States and Europe but the population has many subsets based on mutations of the dystrophin gene.There are currently no treatments for Duchenne patients approved in the United States and in the past  few months, many of the drugs in final stages of development have not been providing much positive news for the Duchenne community.  In January, the FDA did not approve BioMarin’s drisapersen to treat Duchenne patients amenable to exon 51 skipping while the FDA is still reviewing Sarepta’s eteplirsen for that same population. In February, Eli Lilly announced their pivotal Phase 3 study in Duchenne patients failed to meet its primary endpoint. That study was looking at the efficacy of tadalafil in all Duchenne patients. Finally, the FDA recently refused to review PTC Therapeutics’ ataluren is designed to be effective in a 13% subset who have nonsense mutations in the dystrophin gene. And with today's news, many are skeptical that eteplirsen will get approved. However, one thing we did learn today - the patient community around eteplirsen is very load, very organized, and very influential. So, i would not sell your Sarepta stock just yet. The patient community is not finished.

To be continued....

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