FDA Rare Neurodegenerative Disease Grants Program

Clinical Trials Addressing Unmet Needs of Rare Neurodegenerative Diseases

Funds clinical testing research for rare neurodegenerative disorders and therapeutic development.

⚠ This may reflect a past cycle — verify the current call on the funder's site.

The FDA Rare Neurodegenerative Disease Grants Program was established by the ACT for ALS Act of 2021 and has been active since 2022. Administered by the FDA Office of Orphan Products Development, the program funds clinical trials of interventions intended to prevent, diagnose, mitigate, treat, or cure amyotrophic lateral sclerosis (ALS) and other rare neurodegenerative diseases in both adults and children. Unlike the broader Orphan Products Clinical Trials Program, this program was specifically legislated to address the severe unmet need in rare neurodegeneration, and it awards both grants and contracts to public and private entities. The current NOFO is RFA-FD-25-001 (R01 mechanism), submitted through grants.gov. The October 21, 2025 receipt date has passed; no successor NOFO dates are confirmed as of mid-2026, and prospective applicants should subscribe to grants.gov alerts for new RFA-FD-26-XXX postings.

Eligibility is broad: any public or private, domestic or foreign, for-profit or nonprofit entity may apply; federal agencies are excluded. The scope is restricted to rare neurodegenerative diseases, with ALS as the named condition under the ACT for ALS Act; other rare neurodegenerations affecting adults or children also qualify. Mainstream or common neurological indications fall outside the programme scope. Clinical trials must evaluate the efficacy and/or safety of an intervention in support of a new indication or labeling change. As with OOPD's other grant programmes, no per-project award ceiling is published in the available programme pages.

FDA seeks collaborative, efficient, and innovative clinical trial designs under this NOFO, consistent with its broader rare disease strategy. Proposals are expected to articulate the specific unmet need in the target rare neurodegenerative disease population, justify the interventional approach, and present a feasible clinical trial plan. Because the program issues both grants and contracts, applicants should confirm the instrument type when a new NOFO is posted. Teams actively developing rare neurodegeneration therapies should prepare scientific rationale documents and study protocols in advance of the next receipt announcement.