FDA looks to repurposing older drugs for new uses
The US Food and Drug Administration (FDA) is asking for input on repurposing older drugs for new therapeutic uses to address unmet medical needs across a wide range of therapeutic areas.
FDA said that targeting potential new uses, such as a new indication or a new population, for approved drugs can help expedite the availability of treatments by using existing knowledge about the drugs, including their safety profiles.
This request for input was published in a Federal Register. FDA said it is targeting three drug categories: drug candidates where sufficient data may already support a new use; candidates with promising preliminary clinical data from observational studies; and candidates supported by preclinical findings, including outputs from artificial intelligence (AI) and machine learning (ML) tools.
“Too many patients lack effective treatment options, even when promising science exists,” said FDA Commissioner Marty Makary, who reportedly resigned from his position on Tuesday. “Drug repurposing can make better use of available scientific data to deliver effective treatment options for patients in need.”
The notice states that stakeholder efforts to advance drug repurposing, such as workshops and white papers, have focused on challenges and opportunities related to this purpose. Acknowledging the potential public health benefits of drug repurposing, the September 2025 Make Our Children Healthy Again strategy report directed FDA to collaborate with the National Institutes of Health (NIH) to explore using repurposed drugs for treating chronic disease.
During a meeting last May, officials from nonprofit organizations and Janet Woodcock, the former principal deputy commissioner and long-time director of the FDA's Center for Drug Evaluation and Research (CDER), agreed on the necessity for a regulatory framework to facilitate the repurposing of generic drugs for new uses. (RELATED: Repurposing generic drugs: Experts envision new regulatory pathway, Regulatory Focus 12 May 2025)
FDA stated that this initiative builds on existing statutory authorities to update drug labeling by adding new uses for existing products. These authorities include the Best Pharmaceuticals for Children's Act (BPCA) and the Making Objective Drug Evidence Revisions for New (MODERN) Labeling Act of 2020.
Additionally, the initiative builds on Project Renewal, a public health program established by the FDA's Oncology Center of Excellence (OCE). Project Renewal aims to update the labeling for certain older oncology drugs to ensure that the information in the product labeling is both clinically relevant and scientifically current.
FDA is seeking public input from patients, clinicians, public health officials, and researchers regarding priority disease areas and potential drugs for repurposing. The agency wants feedback in the following areas:
- FDA has identified priority areas for chronic diseases, including metabolic disorders, neurodegenerative conditions, women's health issues, men's health issues, substance use disorders, and rare diseases. Do you agree with these priority areas? Are there any additional areas you would recommend for inclusion?
- Which candidates for drug repurposing show the most promise for treating specific medical conditions?
- Are there any new methods for identifying candidates for drug repurposing? What strategies do you think the FDA could employ to facilitate the discovery of new candidates?
The FDA stated in its announcement that public input will assist the agency in evaluating and promoting more opportunities for drug repurposing.
The deadline for submitting comments is 11 June 2026. Respondents should submit comments to https://www.regulations.gov and reference Docket No. FDA-2026-N-4492.
