Stakes Are High Ahead of FDA Panel Vote on ALS Drug

MONDAY, March 28, 2022 (HealthDay News) -- Advocacy groups are pressing U.S. federal regulators to fast-track approval of an experimental drug treatment for amyotrophic lateral sclerosis (ALS), with a decision expected this week.

The push to approve the drug, so far just called AMX0035, is based on partial data from clinical trials and follows the U.S. Food and Drug Administration's controversial approval last year of the Alzheimer's drug Aduhelm. Many of the FDA's own advisors said the costly Alzheimer's drug was unlikely to help patients. But advocacy groups for patients with ALS are pushing hard for approval of AMX0035. They say the need is urgent. AX0035 is an experimental combination drug that has been shown to extend survival for ALS patients.

An FDA advisory committee is scheduled to meet Wednesday to consider a request from AMX0035's maker, Amylyx Pharmaceuticals, to skip the usually required phase 3 clinical trial and approve its drug based on earlier results. The drug company's request is supported by the ALS Association, one of the main advocacy and research groups devoted to finding a cure for this disease.

"The community has been asking for a long time that the FDA approve the drug before that [phase 3] trial is complete," Neil Thakur, chief mission officer of the ALS Association, told HealthDay. "And the reason why is because of a combination of strong clinical benefit and safety data that we've seen for this drug." AMX0035 has been shown in early clinical trials to extend ALS patients' lives by about six and a half months, Thakur said.

The drug also slows disease progression in patients by about 25 percent, James Berry, M.D., director of the Massachusetts General Hospital's Neurological Clinical Research Institute in Boston and a principal investigator in the AMX0035 trials, told HealthDay. "ALS is a disease that doesn't give us a lot of time to sort of stop and consider," Berry said. "We really need to move when we see something that looks this promising without a safety concern and with a very well-run study that shows us both survival and function benefit."

AMX0035 is a combination of two established drug ingredients -- sodium phenylbutyrate and taurursodiol. Sodium phenylbutyrate is sold under the brand name Buphenyl to treat liver diseases, while taurursodiol is a dietary supplement used in ancient Chinese medicine. Some ALS patients already take both drugs. FDA approval would likely compel insurers to cover the treatment.

Until late last year, the FDA had insisted that Amylyx complete ongoing phase 3 trials before seeking approval for AMX0035. In December, the agency relented and began a priority review of the drug application, setting June 29 as the target date for a decision.

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