In reversal, FDA panel recommends controversial ALS drug

An independent FDA panel voted 7-2 to recommend Amylyx Pharmaceuticals' amyotrophic lateral sclerosis drug Sept. 7, about five months after the committee voted 6-4 against the experimental treatment because they couldn't determine if the drug worked. 

The advisory committee said refreshed trial data from Amylyx's ALS drug, named AMX0035, helped determine the vote. The Cambridge-based biotech company's co-CEO, Justin Klee, also said he would withdraw the treatment from the market if a larger trial of 600 people isn't successful. 

"We will do what is right for patients," Mr. Klee said. 

ALS, also known as Lou Gehrig's disease, is a degenerative disease that afflicts about 30,000 Americans, according to the CDC. Patients usually live one to five years after symptoms appear.

If the FDA approves AMX0035 — which could be decided by Sept. 29 — it will be the first ALS drug "that has demonstrated a significant slowing of disease progression and functional decline, as well as extended survival, in a randomized, placebo-controlled clinical trial," Amylyx said in a Sept. 7 news release

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