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Dashing hopes of parents and investors, the US Food and Drug Administration on Thursday released documents suggesting the agency continues to have a highly unfavorable view of a drug used to combat Duchenne muscular dystrophy, a rare disease that causes muscles in boys to stop working and eventually results in death because they can no longer breathe.

In blunt language, agency staffers disputed the analysis and methodology behind crucial data that Sarepta Therapeutics used to bolster its case for approval of its drug, which is called eteplirsen. Notably, the FDA medical reviewers also made a point of rebuking the company for alleging earlier this year that the agency made “key inaccuracies” in a previous assessment.


The fate of the Sarepta drug has been closely watched as a litmus test for an intensifying struggle between the FDA and patient groups that want the agency to take a more expansive view toward approving medicines for unmet medical needs. In recent months, the FDA has rejected two other drugs for DMD, which afflicts about 13,000 children, mostly boys.

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  • The need is indeed great, BAK, but for something that works. I found a bit ago that the price is estimated at $265000/year per patient. Given that the pries are pure fantasy, it’s what the company would like to charge. If only 5,000 out of the 13,000 get the drug at that price (just using the numbers out there), the cost would be $1.3 Billion USD per year. That’s a … high number to essentially run a Phase 4 (well, for this, decent Phase 3) clinical trial.

  • This is an awful thing for any parent to have to deal with, I can’t imagine the agony. I am reminded of another study that had a sample size of 12 children. The results are still being felt. That was the study by Andrew Wakefield, who published the conclusion that MMR Vaccine caused autism. There needs to be a larger sample size and long-term follow up.

    • Your reply frames the dialectic – because this is a BAD disease, any kind of ‘cure’ must be good. Even if it isn’t really a cure and is back-channel supported by people with a profit driver,

  • I don’t know how todays’ meeting will turn out but IMHO the driver is money. Even if the FDA replaces statistics with anecdotal results, however narrowly defined, I forsee a high priced med being prescribed at physician discretion/ coercion for any/every child who has this malady. Hope is pricele$$ but drug$ are not – this is a push to make FDA unlock the money spout. If the price is set at $100/month, I will profoundly apologize.

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