Today we filed public comments with the FDA advisory committee considering Amylyx Pharmaceutical’s new drug application for AMX0035, urging the agency to approve the application.
The comments will be considered by the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee, which will hold a public hearing on March 30.
AMX0035, which is a combination of two compounds that have already been approved, met its primary endpoint in a clinical trial where it slowed loss of function. In a follow-on open label extension study, the drug was shown to extend life by six months compared to placebo.
“The FDA has all the information and regulatory flexibility it needs to approve this therapy today. The FDA has already heard from the ALS community that they cannot wait any longer for approval of a treatment that will have such a on people living with ALS today,” said Calaneet Balas, president and CEO of The ALS Association.
In the formal comments submitted Wednesday, we stated:
While we recognize FDA typically relies on two adequate and well-controlled trials, we implore the Agency to exercise the flexibility described in the FDA’s 2019 guidance on Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products. This guidance specifically allows for the approval of therapies with just one adequate and well-controlled trial in the case of severely debilitating and deadly rare diseases with substantial unmet medical need. Given the important findings and the enormous unmet medical need for people with ALS, we believe the AMX0035 studies conducted to date are sufficient to support approval.
In other words, there is no ethical or scientific justification to delay access to AMX0035 for people living with ALS. AMX0035 complements, and a does not duplicate, all other ALS treatments available. It offers unique benefit to people living with ALS today. Every year of delay in approval will result in thousands of life-years lost.
Calaneet will provide oral testimony at the advisory committee’s March 30 hearing along with Scott Kauffman, chair of the Association’s board of trustees, and board members Larry Falivena and Ken Menkhaus.
