FDA Advisory Committee Recommends Approval of AMX0035

Emily Cerbone Advocacy, Foundation Blog, Home Page

Watch our oral testimony at the FDA Advisory Committee meeting on September 7, 2022.

We are grateful to the FDA Advisory Committee for examining the evidence, listening to the ALS community, and voting to recommend approval of Amylyx Pharmaceuticals’ therapeutic compound, AMX0035.

Amylyx presented new, compelling data analyses on survival. Clinicians, researchers, advocacy groups, and people living with ALS spoke in overwhelming numbers in favor of approval. The reasons are clear: AMX0035 addresses an urgent and unmet medical need. It is safe and effective for treatment of ALS. Trials have shown it can extend survival by 10 to 18 months, and it can slow decline in function by as much as 25%.

AMX0035 should be available to people living with ALS in the United States – and, we hope, it soon will.

Today is a first step. There is a long walk ahead. We need more treatment options for ALS. But this is a moment to be thankful – to Amylyx, to the clinicians, researchers, and advocates who supported this, and especially to the people with ALS who participated in clinical trials at sites including the Les Turner ALS Center at Northwestern Medicine. Many of them are no longer with us. They made this possible. Today’s outcome is their gift to us.

We urge the FDA to act swiftly on this recommendation and provide full approval when it meets on Sept. 29. There is no time to waste.