ORLANDO -- On Saturday, October 8th, more than 100 families living with ALS will have the opportunity to connect with one another for a day of impactful learning and discussions on topics related to the disease. The Symposium will take place at the Rosen Shingle Creek Hotel and Conference Center in Orlando starting at 8 a.m
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.
Earlier this week, The ALS Association filed comments with the Centers for Medicare and Medicaid Services (CMS), urging the agency to provide Medicare coverage for seat elevation systems in power wheelchairs since these systems are a medical necessity for people living with ALS.
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.
Today, we filed comments with the FDA imploring them to ask the right question when it reconvenes its advisory committee in September to consider a new drug application (NDA) for AMX0035.
A committee convened by the Institute for Clinical and Economic Review voted overwhelmingly on August 19 that oral edaravone and AMX0035 provide meaningful clinical benefit to people with ALS and have a positive impact on quality of life, but that neither drug provides sufficient value to justify the presumed cost to insurers.
The House of Representatives passed the Inflation Reduction Act, which includes important health care reforms that will provide financial relief for people living with ALS and their families.
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.
“We are grateful to Cytokinetics for their partnership and transparency, enabling the global research community to leverage these data in their ongoing analyses and research activities,” said Neil Thakur, Ph.D., Chief Mission Officer at The ALS Association.
When the FDA recently accepted Biogen’s new drug application for tofersen, it signaled hope for thousands of people with SOD1 mutations that cause ALS. The FDA has granted priority review to this new drug application and is expected to decide on whether or not to approve tofersen by January 25, 2023.
The researchers focus on an earlier phase of ALS development, which is commonly understood to be a “silent” phase – before symptoms of the disease begin to manifest. Those minor motor impairments in the earlier phase are currently insufficient for a confirmed diagnosis.
The ALS Association filed formal objections with the Institute for Clinical and Economic Review, commonly known as ICER, over their flawed draft report on the cost-effectiveness of AMX0035 and oral edaravone.
People with ALS, caregivers and people at risk of an ALS diagnosis have the opportunity to help the National Institute of Neurological Disorders and Stroke (NINDS) review applications for funding to support research into expanded access.
The Food and Drug Administration has extended the timeline of its review of AMX0035 citing additional data further showing that it is effective at reducing harms associated with the disease.