Sarepta. Why? (opens in new tab)

(science.org)

11 pointsfreditup1y ago3 comments

3 comments

The context here (since the title isn't too revealing), is that the FDA expanded approval for a drug despite essentially unanimous didagreement from the scientists actually analyzing the drug. Derek Lowe, a well-respected pharma blogger, writes about how this was a big mistake and a very bad look for the FDA.

ejang01y ago

"Apparently during the conference call, when [the Sarepta CEO] was asked about why he was so confident [about the Duchenne muscular dystrophy medication's approval], he said that the FDA's CBER head Peter Marks was "very supportive". [...]

Boy, was that the truth. The agency has just granted that use expansion, and it turns out that it was all due to Peter Marks, who completely overruled three review teams and two of his highest-level staffers (all of whom said that Sarepta had not proven its case)."

[The article then talks about how this may be "A positive vote, which marks an undeserved and potentially hazardous victory of emotional rhetoric and relentless patient advocacy over the scientific and medical evidence."]

This is interesting. I didn't know about the influence of the CEO and this narrative around this medications approval/label expansion.

I'm not sure why I'm mentioning this (Devil's advocacy?), some users on HN have also commented that maybe it is good that the FDA approves medications more liberally to give clinicians and patients a chance to experiment with them (if their situations are dire).

Anyways, in this case, it is interesting to think about how much influence one person can have in the FDA.

Sort of related, does anyone remember the approval of aducanumab for Alzheimer's disease? https://www.npr.org/2021/06/11/1005567149/3-experts-have-res...

And now it is discontinued: https://www.ncbi.nlm.nih.gov/books/NBK573062/ This isn't really a good source for the full story, sorry.

All this FDA approval business is much less cut and dry than I would have thought 10 years ago.

jamesboehmer1y ago

I'm seeing all sorts of negative takes on this. But I read one interesting take on reddit from a father of 2 kids with DMD that had a very specific and logical reason for supporting it (other than the treatment helped his kid).

Essentially the efficacy test is a 3 point scale that measures standing and walking. It's not granular enough to get real statistical significance on, and never will be unless another kind of test is defined. So the tests are basically always doomed to fail rigid scrutiny by the FDA.

In the meantime they're getting dramatically effective results, and Peter Marks is bucking the FDA's traditionally conservative stance to make the product available to the few who desperately need it. I'll eat my words if someone finds he was paid off for this. I think is a truly miraculous kind of therapy, and I hope to one day have regularly available gene therapy for my own afflictions.

https://www.reddit.com/r/technology/comments/1dlsset/comment...

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