There’s an urgent need to improve and extend the lives of patients with rare neurodegenerative diseases, and the FDA is hoping a new five-year action plan, which has a specific detailed blueprint for amyotrophic lateral sclerosis (ALS), can help accelerate new medicines.
The broader plan will establish a new FDA Rare Neurodegenerative Disease Task Force, a public-private partnership focused on speeding up the creation of disease-specific science strategies and leveraging ongoing FDA regulatory science efforts to boost scientific advances.
But for ALS, a devastating progressive disorder of the nervous system that causes loss of muscle control, the FDA has a more specific plan to find new ways to quantify disease progression, discover new biomarkers and implement basic sciences discovery as needed.
The FDA will facilitate patient access to new drugs when possible and will promote clinical trial access for diverse populations using digital strategies and decentralized technology. The strategy will also see work done to develop clinical trial strategies to select promising therapies early on, streamline clinical trial operations and reduce the time and cost of drug development.
Patient engagement, public workshops, research projects and coordination across government agencies under the National Institutes of Health (NIH) umbrella will be key to the ALS Science Strategy.
“The effects of rare neurodegenerative diseases are devastating, with very few effective therapeutic options available to patients. We recognize the urgent need for new treatments that can both improve and extend the lives of people diagnosed with these diseases,” said FDA Commissioner Robert Califf, M.D. “To face that challenge and to accelerate drug development, we need innovative approaches to better understand these diseases while also building on current scientific and research capabilities.”
This new strategy is part of the Accelerating Access to Critical Therapies for ALS Act, or ACT for ALS, which was signed into law by President Joe Biden in December 2021. It includes provisions for the Department of Health and Human Services to establish a public-private partnership for neurodegenerative diseases between the NIH, the FDA and one or more outside entities as well as a directive to award grants and contracts to public and private entities for R&D to treat ALS and other rare neurodegenerative disease.
This grant program will be administered by the FDA’s Office of Orphan Products Development to help identify new disease targets and increase the productivity of clinical development.
The ALS community has been particularly active in pushing the FDA to provide access to new treatment options. In March, an FDA advisory committee voted against recommending a therapy under development by Amylyx for approval based on concerns about the single-study application. The meeting was highly emotional—a fact not lost on the committee members—as patients and their caregivers pushed for a chance to try the experimental therapy for their fast-moving condition.
The FDA has yet to rule on the treatment, called AMX0035, but the agency recently pushed the decision out a few months to allow more time to consider the application.