Overview

• FDA chief Martin Makary previewed an upcoming labeling change for leucovorin, saying it could help many children with autism, which has fueled confusion and heightened expectations among families.
• The pathway would apply only to people with low folate in cerebrospinal fluid consistent with cerebral folate deficiency, a subset estimated at roughly 7–30% of autistic individuals depending on measurement.
• Researchers and major autism groups say trials to date are small and heterogeneous, leaving uncertain benefits, unclear dosing, limited pediatric safety data, and specific concerns about use during pregnancy.
• The agency is pursuing a label update for existing generics rather than a new proprietary formulation, a move expected to spur prescribing, expand insurer coverage, and drive business for compounding pharmacies and supplement sellers.
• Clinician-researchers who had discussed a purified, patentable version with federal officials say they were surprised by the rapid shift, while the NIH says it will monitor outcomes and study broader effects with study details still pending.