Drug Repurposing: Often a Lifeline for Patients Living with Rare Diseases

The U.S. Food and Drug Administration (FDA) announced in May 2026 that it was soliciting public comments on its efforts to find new indications for existing FDA-approved drugs to expand treatment options for rare diseases. The FDA specifically requested information on drugs with sufficient evidence to support repurposing to treat other disease states, those with preliminary evidence supporting further study, and those with preclinical data supporting further study (FDA, 2026).