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.
