Agency is particularly focused on new uses to treat chronic or rare diseases
The U.S. Food and Drug Administration today announced it is soliciting input on efforts with respect to drug repurposing to help address unmet medical needs across a range of diseases and conditions.
Identifying potential new uses—such as a new indication or a new population—for FDA-approved drugs can help accelerate the availability of treatments by using existing knowledge about the drugs, including a drug's safety profile. This request for public input is part of a broader FDA initiative to update the labeling of FDA-approved drugs, when supported by sufficient evidence, to ensure that information in the labeling is clinically meaningful for health care providers and patients and scientifically up to date.
“Too many patients lack effective treatment options, even when promising science exists,” said FDA Commissioner Marty Makary, M.D., M.P.H. “Drug repurposing can make better use of available scientific data to deliver effective treatment options for patients in need.”
The FDA is seeking information from patients, clinicians, researchers, and other stakeholders on priority disease areas and potential candidates for drug repurposing, particularly where there are scientific data that could support approval of potential new uses but there appears to be limited commercial incentives to pursue approval of those uses.
The agency is particularly interested in input on disease areas with significant unmet medical need, including metabolic diseases, neurodegenerative conditions, women’s and men’s health conditions, substance use disorders, and rare diseases, as well as other areas stakeholders believe should be prioritized.
Through this docket, the FDA is requesting information on:
- Candidates for drug repurposing where sufficient evidence may already exist to support a potential new use,
- Candidates with promising preliminary clinical data (e.g., data from case reports, case series, observational studies) that may warrant further study, and
- Candidates with promising preliminary preclinical data (e.g., findings from emerging tools such as artificial intelligence and machine learning) that may warrant further study.
The agency is also seeking feedback on innovative approaches to identifying repurposing opportunities as well as barriers that may limit the development or use of repurposed drugs, particularly in cases where there is little or no commercial incentive to pursue labeling changes supported by publicly available scientific evidence.
This effort builds on existing authorities and initiatives that support updating drug labeling when supported by scientific evidence, including the Best Pharmaceuticals for Children Act, the Making Objective Drug Evidence Revisions for New (MODERN) Labeling Act of 2020, and FDA-led initiatives such as Project Renewal, which has updated labeling for several oncology drugs to reflect current scientific understanding. Additionally, the September 2025 Make Our Children Healthy Again strategy report directed FDA to jointly investigate opportunities with the National Institutes of Health to “strengthen the use of repurposed drugs for the treatment of chronic disease, while harmonizing authorization processes through collaborative clinical trial designs to achieve FDA approval.”
Input from the public will help inform how the FDA refines its approach to evaluating and facilitating additional drug repurposing opportunities, including collaborations with federal partners such as the National Institutes of Health and the Centers for Medicare and Medicaid Services.
Interested parties may submit electronic or written comments, data, and information to the docket. Electronic submissions may be made via the Federal eRulemaking Portal at https://www.regulations.gov. Written submissions may be mailed or delivered to the Dockets Management Staff (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
All submissions must include the docket number FDA-2026-N-4492 for “Drug Repurposing for Unmet Medical Needs; Request for Information.”
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