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.
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.
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.
The ALS Association, in collaboration with ALS Finding a Cure, recently awarded $400,000 to support research into the role SARM1 gene mutations play in the development and progression of ALS, with the ultimate goal of using an improved understanding of SARM1 to develop new treatments.
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.
The ALS Association, thanks to support from the Greater New York Chapter, has awarded $525,000 to four projects through its Milton Safenowitz Postdoctoral Fellowship program, which supports the development of new scientists in the field of ALS research.
Biogen and Ionis Pharmaceuticals have decided to discontinue research testing the investigational drug BIIB078 after a phase 1 clinical trial did not meet any secondary endpoints on efficacy and did not demonstrate clinical benefit.