FDA Holds Public Advisory Meeting to Review Application for Drug to Treat ALS

The U.S. Food and Drug Administration held an advisory committee meeting on Wednesday to review concerns over evidence from Amylyx Pharmaceuticals on the ability of a drug, AMX0035, to treat amyotrophic lateral sclerosis.

ALS is a rapidly progressive and fatal neurodegenerative disease that can lead to the loss of voluntary movement and the development of difficulty in swallowing, speaking and breathing.

There are approximately 6,000 new cases of ALS reported in the U.S. each year, with a large share of those cases in people aged 40-70. 

There is no cure for ALS, but the FDA has two approved therapies, riluzole and edaravone, which improve quality of life issues such as cramps and spasticity.

The application for AMX0035 to the FDA included one double-blind, placebo-controlled Phase 2 study of 137 patents with ALS. 

In a briefing document sent prior to the committee meeting, the FDA expressed concerns that the “data may not be adequate … and encouraged the applicant to urgently begin work on a Phase 3 study to confirm the findings.”

The committee discussion focused on study conduct issues and detailed statistical considerations of the analyses of the study’s endpoints, the persuasiveness of the trial submitted and the seriousness of the disease.

“No final decision has been made on approvability. … As we conduct our reviews, we continue to keep in mind the context that ALS is a rare and devastating disease, with an enormous unmet medical need,” said Teresa Buracchio, director of the Division of Neurology 1 at the Office of Neuroscience, Office of New Drugs, CDER, FDA, during the committee meeting. 

“However … it is legally required for [the] FDA to ensure that drugs are both effective and safe for approval,” continued Buracchio.

A decision from the FDA is expected by June 29.

Alexa can be reached at [email protected]