FDA approves ALS drug based on 1 phase 2 trial

Months after an FDA panel voted against Amylyx Pharmaceuticals' amyotrophic lateral sclerosis drug, a second panel reversed the decision in a surprise vote before the agency approved the controversial ALS treatment Sept. 29. 

In late March, an FDA advisory committee voted 6-4 against Amylyx's treatment, which is now named Relyvrio, because they said the company's clinical trial didn't prove the drug's effectiveness. In a bid to prove the treatment works, Amylyx then submitted more data from the phase 2 trial with 137 ALS patients. 

In the refreshed trial — which was partly funded by the ALS Ice Bucket Challenge, according to Amylyx — people who took Relyvrio lived about 10 months longer than those who didn't, the company said May 5. The FDA met a second time to discuss the new results before voting 7-2 in favor of it on Sept. 7. 

ALS, which affects nearly 30,000 people in the U.S., kills patients within one to five years of symptoms appearing, according to the CDC. There is no cure for the degenerative disease but there are multiple drugs currently on the market. 

With another trial underway involving 600 people, the Cambridge, Mass.-based company's CEO, Justin Klee, said he would withdraw the drug from the market if the trial doesn't confirm the most recent results. 

"Today's FDA approval of Relyvrio is an exciting milestone for the ALS community and is a major step toward achieving our mission to one day end the suffering caused by neurodegenerative diseases," Mr. Klee said in a Sept. 29 statement.

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