The US House of Representatives voted almost unanimously on Wednesday evening (423-3) to pass a bill that will provide $500 million over five years to certain small drugmakers to cover the costs of R&D and to expand access to patients not eligible for clinical trials for potential amyotrophic lateral sclerosis drugs. Rare neurodegenerative diseases, like ALS, have been historically very difficult to treat and to develop treatments for. But this bipartisan bill, introduced by Rep. Mike Quigley (D-IL) and dozens of cosponsors, will provide $100 million for each of fiscal years 2022 through 2026 to help HHS award grants to facilitate the development and access to ALS drugs intended to prevent, diagnose, mitigate, treat, or cure the disease.
ALS has proven to be a graveyard for drug developers so far, with Biogen’s ALS drug tofersen the latest casualty in Phase III fails.
But a windfall of funding from Congress for ALS drug development, which still has to make its way through the Senate and past President Joe Biden, comes at a critical moment, when the FDA’s accelerated approval for Biogen’s controversial Alzheimer’s drug became a spot of contention for the ALS community. Unlike Biogen, Amylyx’s ALS drug was required to run another study to confirm efficacy despite hitting the primary endpoint in a Phase II trial, and meeting some of the stipulations laid out in the FDA’s 2019 guidelines for new ALS treatments.
Sen. Mike Braun (R-IN) in June publicly pressed acting FDA commissioner Janet Woodcock in a hearing to ‘pick up the pace, a bit,’ for ALS drug development. Responding to Braun’s questions about expanding the same flexibility to ALS that the FDA showed in Alzheimer’s, Woodcock said developers and researchers still needed to understand the disease better.
But five months after the FDA asked for a Phase III study to confirm those positive Phase II results, the agency has apparently reversed course. Amylyx will be submitting its ALS drug for US approval after meeting with regulators last month, the company said in September.
This latest bill could provide a boost to companies similar to Amylyx. It would also establish and implement a Collaborative for Neurodegenerative Diseases between the NIH and FDA.
Not later than 6 months after the law is enacted, HHS will also have to come up with an action plan describing how the NIH and FDA will craft a 5-year plan to foster the development of safe and effective drugs that improve the lives of people living with rare neurodegenerative diseases as quickly as possible, and facilitate access to investigational drugs.
The only three members of the House to vote against the bill were Republicans Ralph Norman of South Carolina, Thomas Massie of Kentucky, and Chip Roy of Texas.
https://endpts.com/house-almost-unanimously-passes-bipartisan-bill-to-pump-500m-into-als-drug-rd/
