The FDA cleared leucovorin (Wellcovorin) for a specific, genetic form of cerebral folate deficiency (CFD‑FOLR1), reversing earlier public signals that the agency might greenlight broader use for autism. Agency reviewers concluded data support the rare‑disease indication but do not substantiate generalized autism claims. The approval comes amid high-profile political attention and a surge in off‑label prescriptions after U.S. officials publicly touted the molecule for autism. Regulators emphasized the narrow genetic population that benefits from the drug and warned against extrapolating to broader autism diagnoses without robust clinical evidence. The decision underscores tensions between rapid policy-driven endorsements and traditional evidence standards; clinicians, payors and patient groups will monitor prescribing behavior and potential misalignment between approval language and off‑label demand.