The ALS Association spent over $2 million helping fund the development and clinical trial of AMX0035. When the results of that trial showed it was safe and effective in treating ALS, the ALS Association led an advocacy campaign to push the FDA to approve the drug. After two years of advocacy, the FDA finally approved AMX0035.
The ALS Association, the country’s largest nonprofit committed to making ALS livable and finding a cure, today celebrated the Food and Drug Administration’s (FDA) approval of AMX0035, a new treatment for people living with amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease. The Association invested $2.2 million of funds raised through the 2014 ALS Ice Bucket Challenge into the development and trial of AMX0035, and led the years-long advocacy campaign that pushed the FDA to approve the treatment prior to completion of an ongoing phase 3 trial.
We strongly disagree with ICER’s final report on new ALS drugs, which may result in people living with ALS being unable to access life-extending treatments. ICER’s flawed conclusions were based on their discriminatory methodology, as the National Council on Disability has documented.
An FDA advisory committee voted overwhelmingly (7-2) to support approval of AMX0035 for the treatment of ALS. A phase 3 clinical trial will continue to test the effectiveness of AMX0035.
We talked with Dr. John Kalambogias, postdoctoral fellow from Columbia University, to learn more about his research focused on dysfunction and degeneration of corticospinal tract neurons in ALS mouse models.
ICER’s review process now moves to a public hearing of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), which ICER defines as a core program comprised of health care officials from throughout the Midwest.
New long-term analysis published in the Journal of Neurology, Neurosurgery & Psychiatry showed AMX0035 was effective at reducing a variety of harms associated with ALS during the previously published Phase 2 Centaur trial.