Overview
- The FDA has begun updating leucovorin’s label to note it can improve certain symptoms in adults and children with cerebral folate deficiency, according to a Federal Register notice.
- The action applies to a subset of autistic people with low cerebrospinal-fluid folate rather than all autistic individuals, with estimates of eligibility ranging from about 7% to 30% depending on measurement.
- President Trump and FDA commissioner Martin Makary highlighted the move at the White House, prompting objections from leading researchers and self-advocates who criticized overstated claims and stigmatizing rhetoric.
- Clinicians report increased off-label prescribing and interest from compounding pharmacies even as experts note uncertain dosing guidance, unclear duration of use, and limited pediatric safety data.
- The NIH plans to monitor outcomes and explore broader effects, and observers expect the label change to encourage insurer coverage while scientists call for larger, rigorous trials to test efficacy.