On September 15, Amylyx Pharmaceuticals announced its intention to submit a New Drug Application to the FDA for AMX0035 for the treatment of ALS.
If the New Drug Application is successful, AMX0035 would be only the third drug approved by the FDA for the treatment of ALS.
Les Turner ALS Foundation’s CEO Andrea Pauls-Backman is grateful to the clinical trial participants and stresses the importance of the New Drug Application: “The ALS community has waited a very long time to see a compound make a meaningful difference in slowing the progression of ALS and extending life. Thankfully, AMX0035 appears to do both. We are grateful to the 137 participants who were part of the Phase 2 trial and to Amylyx for its unwavering commitment to the ALS community. In the meantime, our Les Turner ALS Center at Northwestern Medicine will commence enrollment later this fall in the Phase 3 trial of AMX0035, as part of a 600-person international population enrollment.”
Senda Ajroud-Driss, MD, Associate Professor of Neurology and Director of the Lois Insolia ALS Clinic at the Les Turner ALS Center at Northwestern Medicine, and site investigator for the clinical trial, is also hopeful: “The results of the AMX0035 Phase 2 trial and the open-label extension are very encouraging. For people living with ALS, extending life by 6 months is amazing.”
In addition to the importance of the New Drug Application, Andrea also emphasizes the urgency: “We encourage the FDA to move quickly on the New Drug Application and for Amylyx to commence its expanded access to program (EAP) for AMX0035 as soon as possible. The ALS clock waits for no one.”
Along with the urgency needed, extensive collaboration is also critical: Andrea says, “As decades of research have shown us, the clearest path to finding meaningful therapies for ALS is through cooperation between ALS researchers, clinicians, patient advocates, and pharmaceutical companies combined with openness and flexibility on the part of regulators. Every clinical trial needs an Expanded Access Program and an Open-Label Extension to improve access to people with ALS who may not be eligible for specific drug trials. The Les Turner ALS Foundation has continuously pushed our legislators to pass both the ACT for ALS and the Promising Pathway Act to accelerate neuromuscular drug development for all Americans affected by debilitating neurodegenerative diseases.”
In working together as a community with our ALS support team and continuing to advocate for ALS drug approval, progress is made. Even the smallest amount of progress makes a difference. As Dr. Ajroud-Driss says, “Every little step counts and every drug that gets approved is a step in the right direction. The AMX0035 New Drug Application is well designed, so I’m crossing my fingers.”
Read more about the importance and need for faster ALS drug approvals by the FDA in our blog post at: https://lesturnerals.org/fda-news/