Sketchy Data
ABC News reports that a much-debated drug for Lou Gehrig’s disease won U.S. approval Thursday. The FDA approved it with sketchy data. ABC News reported that it was a long-sought victory for patients that is likely to renew questions about the scientific rigor behind government reviews of experimental medicines.
The FDA approved Amylyx Pharmaceuticals based on results from one small, mid-stage study in which patients with the debilitating disease appeared to progress more slowly and survive several months longer.
The FDA usually approves a drug after two large studies or one with “very persuasive” survival results.
“This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” FDA’s neurology drug director Dr. Billy Dunn said in a statement.
The FDA’s internal scientists rejected the drug based on “borderline” and “not persuasive” results.
Outside advisors agreed. Then the panel was brought back, and they changed their minds.
It’s approved.
The company will run a follow-up clinical trial and if it is unsuccessful, the company will take it off the market.
The treatment will be called Relyvrio in the US and is already conditionally approved in Canada. The Food and Drug Administration said Thursday in a statement. “Given the serious and life-threatening nature of ALS and the substantial unmet need, this level of uncertainty is acceptable in this instance,” the agency said. The shares rose 9.8% Friday in trading before US markets opened., Bloomberg reported.
I think ALS is a horrific terminal illness. It destroys the nerve cells that control all the body’s functions within a few years. It slowly tortures people to death. If people know the risks, perhaps it’s worth it, unlike the COVID vaccine. What do you think?
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