FDA rare neurodegenerative disease grants program

Key facts

• The program began with the ACT for ALS (Public Law 117-79), signed 2021-12-23; Section 5 directs FDA to award grants and contracts to public and private entities for ALS and other rare neurodegenerative disease research and development.
• Section 7 of the Act authorizes 100 million USD per fiscal year for fiscal years 2022 to 2026, covering the whole Act rather than Section 5 alone; Section 5 itself has no stated sunset.
• FDA runs it through the Office of Orphan Products Development, as a track alongside the Orphan Products Grants Program, whose clinical trials grants have operated since 1983.
• To qualify, a condition must be rare under 21 USC 360ee and show progressive neuronal loss in the central or peripheral nervous system; primarily muscle diseases, secondary neuronal loss, acute non-progressive conditions and static neurodevelopmental conditions are excluded.
• FDA's definition page and program page do not mention prion disease and list no eligible diseases; examples named on the program page are ALS, Cockayne syndrome, ataxia telangiectasia, MPS III, familial dysautonomia and Niemann-Pick C.
• The awards page lists new awards of about 6.93 million USD in FY2022, 16.9 million in FY2023, about 9.1 million in FY2024, about 10.6 million in FY2025 and at least 6.5 million in FY2026, roughly 50 million USD in disclosed totals (my own sum, a floor).
• Recent individual awards: 5 million USD for a CADASIL retinal biomarker study (FY2024), 3.6 million USD for atipeksen in ataxia telangiectasia (FY2025), and 2.5 million USD for a phase 1 amlodipine study in myotonic dystrophy type 1 (FY2026); each runs 2 to 4 years.
• Forecast FOR-FD-25-006 (studies addressing rare neurodegenerative diseases including ALS) expects 10 awards, has no dates or award size, and was last updated 2026-05-13.
• Forecast FOR-FD-25-001 (clinical trials, R01) shows an award range of 650,000 to 900,000 USD with unclear min/max labelling, no dates, last updated 2026-05-13.
• Both forecasts were first posted 2024-08-14, are open to universities, nonprofits, small and large businesses and governments, and are discretionary grants without cost sharing.
• FDA's funding page (current as of 2026-04-14) lists only one neurodegenerative NOFO, RFA-FD-25-001, with a single receipt date of 2025-10-21, which has passed; the general rare disease clinical studies NOFO RFA-FD-25-020 has receipt dates 2026-10-20 and 2027-10-19.
• In October 2022 FDA announced 19 new grants and two contracts worth over 38 million USD across the broader Orphan Products Grants Program, reported by trade press.

Timeline

1. 1983FDA Orphan Products clinical trials grants program begins.
2. 2021ACT for ALS signed into law as Public Law 117-79, creating the rare neurodegenerative disease grant authority in Section 5.
3. 2022FDA announces the first round of awards tied to the Act, including two contracts for ALS-related work.
4. 2024Grants.gov forecasts FOR-FD-25-006 and FOR-FD-25-001 first posted.
5. 2025Receipt date for NOFO RFA-FD-25-001 (rare neurodegenerative clinical trials).
6. 2026FDA funding opportunities page last reviewed, showing no open RNDD call.
7. 2026Both Grants.gov forecasts last updated, still without dates.
8. 2026Final fiscal year end of the Act's 100 million USD annual authorization (FY2022 to FY2026).