ALS Drug Relyvrio Fails in Large Trial, Raising Questions About FDA Approval Standards for Devastating Diseases

ALS drug Relyvrio, approved by the FDA despite limited evidence, failed in a large clinical trial showing it does not work. The failure raises questions about the FDA's flexible approval process for treatments of devastating diseases like ALS.

Relyvrio was developed by startup Amylyx Pharmaceuticals, founded by two college students. It won FDA approval based on a small 137-patient trial showing slower disease progression.

The FDA has used flexibility to approve Alzheimer's, ALS, and Duchenne MD drugs lacking typical solid proof of efficacy. Critics argue this lowers standards and reinforces pressure to approve more marginal therapies.

Emotional patient advocacy heavily influenced Relyvrio's approval, with patients begging the FDA committees for the chance to live longer even if benefits were uncertain.

After Relyvrio's failure, Amylyx lost most of its $1 billion valuation. But its founders say they took the right path and remain dedicated to helping ALS patients who still need solutions.