FDA Opens Docket FDA-2026-N-4492 Soliciting Drug Repurposing Candidates for Metabolic, Neurodegenerative and Rare Diseases
The agency is asking patients, clinicians and researchers to nominate FDA-approved drugs that could be relabeled for new indications, including those backed by case reports or AI-generated preclinical evidence.
Overview
The U.S. Food and Drug Administration on May 11 announced it is soliciting public input on drug repurposing as a way to address unmet medical needs, opening docket FDA-2026-N-4492 for stakeholders to nominate FDA-approved drugs that could be relabeled for new indications or new patient populations, according to the agency’s press announcement.
What the FDA is asking for
The request for information targets three categories of repurposing candidates, per the FDA: candidates where “sufficient evidence may already exist to support a potential new use”; candidates with promising preliminary clinical data such as case reports, case series, and observational studies; and candidates with promising preliminary preclinical data, including findings from emerging tools such as artificial intelligence and machine learning.
The agency is particularly interested in disease areas with significant unmet medical need, naming metabolic diseases, neurodegenerative conditions, women’s and men’s health conditions, substance use disorders, and rare diseases, the FDA said, and is also inviting nominations of additional priority areas.
Beyond candidate identification, the docket is also collecting feedback on 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, according to the FDA.
Commissioner’s statement
FDA Commissioner Marty Makary, M.D., M.P.H., said in the announcement, as published by the FDA: “Too many patients lack effective treatment options, even when promising science exists. Drug repurposing can make better use of available scientific data to deliver effective treatment options for patients in need.”
Existing programs the request builds on
The FDA framed the request as part of a broader effort to update the labeling of FDA-approved drugs when supported by sufficient evidence, according to the press announcement. The agency cited 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 the FDA said has updated labeling for several oncology drugs to reflect current scientific understanding. Project Renewal, run out of the FDA’s Oncology Center of Excellence, was described in a 2021 paper in Clinical Cancer Research by Paul G. Kluetz and colleagues as a collaborative initiative to engage the oncology community to update product labeling for older oncology drugs, as indexed on PubMed.
The FDA said input from the public will help inform how it 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, per the announcement.
How to submit
All submissions must include the docket number FDA-2026-N-4492 for “Drug Repurposing for Unmet Medical Needs; Request for Information,” the FDA stated. Electronic submissions may be made via the Federal eRulemaking Portal at https://www.regulations.gov.
What we don’t know
The FDA’s announcement does not on its face specify the length of the comment period or the deadline for submissions, nor does it commit to any specific regulatory action that will follow the comment window. The agency also did not name initial repurposing candidates or pre-identify any drugs it intends to evaluate first; the docket is structured as a solicitation of nominations rather than a fixed agenda.
The Federal Register notice corresponding to docket FDA-2026-N-4492 had not appeared in the Federal Register’s online search results at the time of the FDA’s press announcement; the docket and any companion regulatory text are expected to follow on the Federal eRulemaking Portal.