The ALS Association submitted comments to the FDA’s Central and Peripheral Nervous System Advisory Committee, urging it to recommend approval of tofersen for people with ALS linked to a SOD1 gene mutation. The committee will meet virtually on March 22 to review data associated with Biogen’s new drug application.
Biogen Inc. announced that the U.S. Food and Drug Administration extended the review period of the promising gene therapy treatment Tofersen to consider additional data. The initial review period, which was announced in July 2022, was scheduled to conclude in January 2023. The review will now be extended to April 25, 2023.
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.
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.
The ALS Association endorses the strengthened Accelerating Access to Critical Therapies for ALS Act (ACT) for ALS Act (H.R.8662/ S. 4867) as an important step in bringing promising new treatments to people with ALS as quickly as possible. The bill accelerates the fight against ALS by authorizing $100 million for ALS research and creating the first federal entity explicitly charged with developing treatments for neurodegenerative diseases.
The ALS Association strongly supports initiatives to enable people with ALS to access promising treatments as soon as possible, including prior to FDA approval. Our primary goal is to facilitate the development of effective treatments and help support delivery to everyone with ALS as soon as possible.