Pressured by patients, FDA will review an ALS drug with modest data
The agency may soon approve another drug for a deadly neurodegenerative disease based on partial data that’s being debated by experts.
The Food and Drug Administration meets this week to review a closely watched drug for ALS, or Lou Gehrig’s disease, following months of lobbying by patient groups and congressional lawmakers.
The drug from Amylyx Pharmaceuticals showed modest benefits in one small study and the FDA told the company last April to run a larger one before seeking approval.
But that delay sparked pushback from patients, their families and members of Congress.
In September the FDA reversed course and agreed to review the drug based on the single study.
Experts say it's the latest example of patients exerting political pressure to push drugs through the FDA approval process.