FDA Advisors Cast Doubts About Amylyx's Experimental ALS Drug

Neuroscience experts advising the FDA have found that the study did not provide sufficient evidence that Amylyx Pharmaceuticals Inc’s experimental amyotrophic lateral sclerosis drug works.

Their 6-to-4 vote sets up a new test for the FDA as it considers approving the latest experimental drug promising to slow the march of the debilitating neurodegenerative disease. The FDA has a June 29 deadline for making a decision on the Amylyx drug.

Some of the advisors who cast votes after hearing an emotional plea from ALS patients and their families said they faced a difficult decision but were asked about the results of a lone trial of the drug.

The FDA is under pressure from some patients with Alzheimer's ALS and other neurodegenerative diseases to approve drugs that show promising signs for treating these conditions.  

However, the FDA has also been criticized for relaxing its standards and not scrutinizing experimental drugs, resulting in the sale of expensive new drugs that may not help patients.

The issue came out with the recent approval of a Biogen Inc Alzheimer’s disease drug called Aduhelm, about which some critics have raised doubts and which also has serious side effects.

According to a document published online by the FDA, the FDA has asked Amylyx to conduct a second study before first applying for approval. The FDA later changed his mind and told Amylyx to go ahead and apply.

The shares of Amylyx fell 36% on Monday after the staff’s review of the drug application was released by the FDA ahead of the advisory panel’s meeting. According to the document that FDA posted online, the agency’s staff concern included methods that Amylyx used to analyse the study results and account for subjects who passed away.