Overview

• FDA chief Martin Makary announced an upcoming approval, touting leucovorin as potentially helping large numbers of children, though the plan targets only autistic people with low cerebrospinal-fluid folate.
• Researchers emphasize that efficacy has not been established, citing small, heterogeneous trials that relied on subjective outcomes and enrolled as few as 48 to about 80 children.
• The proposed authorization would apply to an estimated 7–30% of autistic individuals, depending on how low folate levels are measured.
• The administration says NIH will monitor outcomes and explore broader effects, yet no study designs, timelines, or prescribing guidance have been released.
• Clinicians report rising inquiries and confusion from families, as some seek prescriptions despite limited availability and scant pediatric safety data, while a prescribing advocate, Dan Rossignol, says he has discussed the drug with the White House.