Earlier today the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 6 to 4 against recommending AMX0035 for approval to treat people with ALS. It’s important to note that the Advisory Committee’s views are not binding on the FDA. Following the vote, the ALS Association called on the FDA to take into account the strong safety profile of AMX0035, as well as the serious unmet medical need of people living with this devastating condition, and approve the drug for clinical use.
It is on us—those who have experienced this disease firsthand, those of us who are living with the disease, those who are serving as caregivers—to determine the value and quality of life with ALS. We need to stand up and object to discriminatory assessments that overlook the most important things that give life value.
The ALS community has the opportunity to encourage The Food and Drug Administration (FDA) to approve Amylyx Pharmaceutical’s new drug application for AMX0035.
The Institute of Clinical and Economic Review, commonly known as ICER, has opened a review of AMX0035 to determine the cost-effectiveness of the drug. The ALS Association is committed to making sure ICER’s review does not discriminate against people with ALS and that its analysis does not prevent people with ALS from accessing promising treatments.
The multidisciplinary care model brings together a team of health care professionals specially trained to address the needs of people living with ALS, allowing them to receive care from each discipline they need during a single visit. The ALS Association collaborates with experienced ALS clinicians across the country to help ensure people living with ALS have access to this type of specialized care.
Amylyx recently filed a New Drug Application for AMX0035, a promising new drug that has proven safe and effective at slowing progression of ALS and extending the life of people living with the disease. The ALS Association has called on the FDA to approve the application with urgency.
After Amylyx’s announced that it intends to file a new drug application for AMX0035, The ALS Association immediately called on the U.S. Food and Drug Administration to approve the treatment for all people with ALS as soon as possible. Connecting ALS talked to the team at Amylyx to learn about the path ahead for access to AMX0035.
Dr. Kuldip Dave, vice president of research at The ALS Association, recently discussed the science of Tregs on Connecting ALS. A transcript of that discussion has been edited and shortened below.