The U.S. Food and Drug Administration today announced it is soliciting public input on drug repurposing — identifying new uses for already-approved drugs — to help address unmet medical needs across a range of diseases and conditions.
The agency is particularly focused on chronic and rare diseases where treatment options are limited and commercial incentives for traditional drug development may be insufficient.
Faster Paths to Treatment
Repurposing FDA-approved drugs for new indications or new patient populations can significantly accelerate treatment availability by leveraging existing knowledge about a drug’s safety profile. This request for public input is part of a broader FDA initiative to update drug labeling when supported by sufficient evidence, ensuring labeling remains scientifically current and clinically meaningful.
“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.”
Priority Disease Areas
The FDA is seeking input from patients, clinicians, researchers, and stakeholders 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 any other areas stakeholders believe should be prioritized.
Three Categories of Candidates
The agency requests information on drug repurposing candidates falling into three categories: drugs where sufficient evidence may already exist to support a potential new use, drugs with promising preliminary clinical data — such as case reports, case series, or observational studies — warranting further study, and drugs with promising preclinical data from emerging tools like artificial intelligence and machine learning that may warrant further investigation.
Building on Existing Initiatives
This effort builds on established authorities and programs, including the Best Pharmaceuticals for Children Act, the MODERN Labeling Act of 2020, and FDA-led initiatives such as Project Renewal, which has already updated labeling for several oncology drugs to reflect current science.
The September 2025 Make Our Children Healthy Again strategy report directed the FDA to jointly explore opportunities with the National Institutes of Health to strengthen the use of repurposed drugs for chronic disease treatment while harmonizing authorization processes through collaborative clinical trial designs.
Input from the public will help inform how the FDA refines its approach to evaluating and facilitating repurposing opportunities, including collaborations with the NIH and the Centers for Medicare and Medicaid Services.
Source: FDA Press Release
