The U.S. Food and Drug Administration approved Wellcovorin (leucovorin) for cerebral folate deficiency caused by pathogenic variants in the folate receptor 1 (CFD‑FOLR1), while explicitly declining broader approval for autism spectrum disorder. The agency based its label expansion on case reports and mechanistic literature rather than randomized trials, a route that prompted public and political attention. GSK supplied historic data for the submission; FDA reviewers said evidence supported treatment of the specific genetic syndrome but was insufficient to endorse leucovorin as a therapy for autism. FDA statements and subsequent reporting highlighted a mismatch between earlier White House and agency messaging and the final regulatory decision. The decision immediately affected prescribing and access patterns: off‑label prescriptions surged after political endorsements, raising concerns among clinicians about supply and appropriate use. Regulators and clinicians now face the operational challenge of clarifying which patients will benefit and how to manage increased demand for a drug with limited trial data.