FDA Formally Approves Leucovorin for Rare Folate‑Transport Disorder, Rejects Autism Use for Now
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The FDA formally approved generic leucovorin for children and adults with an ultrarare genetic condition that blocks folate transport to the brain (affecting fewer than 1 in a million), but said there is not yet enough evidence to support its use for autism and narrowed its indication to only that mutation. The decision follows the retraction of a key autism study and a White House‑driven surge in pediatric prescriptions last fall, and comes amid shortages that prompted the FDA to allow imports while original manufacturer GSK does not plan to relaunch its branded version.
FDA and Drug Regulation
Autism and Public Health Policy
FDA Drug Approvals and Autism Treatments