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A federal advisory panel voted Monday that a drug from Sarepta Therapeutics was not effective for treating Duchenne muscular dystrophy, a rare and fatal muscle-wasting disease. About 13,000 children, mostly boys, are afflicted.

The vote came after a daylong session punctuated by emotional pleas from dozens of parents and their children, some of whom appeared in wheelchairs, to describe how the Sarepta drug, called eteplirsen, made a substantial difference in clinical trials. Their testimony was balanced by presentations from US Food and Drug Administration staff who took a dim view of the Sarepta trial data.


The panel vote, however, is not the last word. The agency must now determine by May 26 whether to follow the recommendation.

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  • A placebo control trial cannot be done with this drug. The cat is out of the bag. At 24 weeks effects can be seen. They (Boys) would know who’s “on Drug” because of improved ADL’s, trial then no is longer placebo blinded.

    If They want a 24 week placebo trial ok, after that it’s no longer a controlled placebo trial. That is what happen, ethical vs unethical FDA trial. It was no longer about absolute science.
    If you want to prove a drug by unethically keeping off drug until a wheelchair is needed fine, NOT for Children. Adults maybe, they have a legal choice, not 7-9 year old children. It’s unethical now. They know the cause and effects. No way to keep it blinded. FDA foot-dragging 4 years at first ok then refusing natural history studies. Then Farkas playing games with his ego at stake to prove his point that only big placebo trials should cross his desk.

    He should change or resign, no DMD drug maker is going to trust him again.

  • Testimonies of Doctors and many of the boys as well as study results showed this treatment was working. Why deny these boys this treatment when right now they have nothing. Each day they lose more muscle use. There isn’t any time to be wasted. GIVE THEM THE DRUG. They have nothing to lose by getting it and much to lose by being denied.

    • Have faith, Carol. Here is where all of the back room wheeling and dealing behind closed doors will occur between FDA and the company. I have the personal experience of my company’s product being flat out rejected on safety grounds by Advisory panel 20 years ago and lo and behold it was approved by FDA one month. Of course their is no written record but certain pressures were brought to bear on certain individuals. FDA is not above such back door maneuverings, I’ve seen with my own eyes, so don’t lose faith.

  • What this panel has done to these children is unconcinable. I understand these parents plite. I had a brain injured child. If there was any medication available in this world that could have helped her, I would have done every thing within my power to get it for my child. How can this panel of people make a decision like this? I would like to know how to get in touch with this panel or parents of these children. I would help in the fight to get this medication approved.

  • You saved the best for last “Unfortunately, the data do not meet a scientific standard of evidence of effectiveness,” she told the panel. “These boys and their families deserve better.”
    That said, given the context, “… It’s advocacy pressure of the sort that I’ve not seen since the days of HIV and AIDS. And that can make the difference here.”
    If the FDA caves to sentiment and sophistry over science, what will the unintended consequences be? Other than stacks of cash for the manufacturer and it’s investors?

    • It seems though that it also wasn’t conclusive that it didn’t work. It seemed to work, just anecdotally, right? They needed more patients in the study to power it better statistically. It can be hard to recruit for studies like this with so few patients out there who meet the eligibility criteria. Might be a good tool for docs to have who want to try it with their patients.

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