Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)
Grant Opportunity Analysis
The Department of Health and Human Services, through NIH and NINDS, is seeking cooperative agreement applications to support scientific research using expanded access data for investigational drugs or biological products for amyotrophic lateral sclerosis (ALS). The work focuses on intermediate-size ALS patient populations who are not eligible for ongoing clinical trials, with the goal of advancing understanding of ALS and informing prevention, treatment, and new therapies. Eligible applicants must be clinical trial sites participating in a phase 2/3 or phase 3 efficacy trial sponsored by a U.S.-based small business, and applications must align with FDA expanded access requirements, including submission of an intermediate EA IND by the application date and FDA approval before funding; foreign organizations and foreign components are not eligible. The closing date is November 10, 2026, with an estimated total program funding of $40 million and about four awards, and questions may be directed to NINDSALSMailbox@mail.nih.gov.
Eligible Applicants
Refer to Section III. Eligibility Information in the NOFO for additional information on eligibility.Eligible applicants must be clinical trial sites that participate in a phase 2/3 or phase 3 efficacy clinical trial (designed to provide pivotal data to support a marketing application) supported by a small business concern that is the FDA-designated sponsor of a drug or biological product which is the subject of an IND under section 505(i) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) to prevent, diagnose, mitigate, treat, or cure ALS. The definition of a small business concern can be found in section 3(a) of the Small Business Act (15 U.S.C. 632(a)).Foreign Organizations/International CollaborationsNon-domestic (non-U.S.) Entities (Foreign Organizations) are not eligible to apply.Non-domestic (non-U.S.) components of U.S. Organizations are not eligible to apply.Foreign components, as defined in the NIH Grants Policy Statement, are not allowed.