FDA Declines Approval of Leucovorin for Autism, Clears It Only for Rare FOLR1 Disorder
Lead: In March 2026, the U.S. Food and Drug Administration declined to expand approval of the generic drug leucovorin for use in treating autism, instead approving it only for a rare genetic condition, cerebral folate deficiency caused by FOLR1 mutations in adults. The decision follows high-profile promotion of the drug by FDA Commissioner Marty Makary … Read more