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.
Follow-up analyses from the Phase 3 Valor study and the open-label extension study of tofersen, an investigational antisense drug, showed that the drug was effective in slowing down progression of ALS in people with SOD1 mutations.
Every year, hundreds of people with ALS, their caregivers, friends and family come together to discuss progress in the fight against ALS and how we advance ALS research, accelerate the drug development process and make it possible for people with ALS to live their lives as they want by engaging policymakers to support the ALS community. Registration is now open for our annual advocacy conference, which will be held June 14 -16 from 3:00-4:30 p.m. ET. Due to visitor restrictions at the U.S. Capitol Complex, the event will be virtual again this year.
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.