FDA Approves Leucovorin for Rare Genetic Condition Linked to Autism Features
The FDA has approved leucovorin for cerebral folate deficiency with FOLR1 gene variant, not for autism broadly. Agency officials cited insufficient data for broader autism efficacy despite previous hype. Prescriptions for the drug surged 71% among children following earlier promotional statements.
The U.S. Food and Drug Administration has expanded approval for the drug leucovorin, but specifically for patients with a rare genetic condition rather than for autism broadly. The agency approved leucovorin for use in adults and children with cerebral folate deficiency who have a variant in the folate receptor 1 gene, marking the first treatment for this rare condition which shares symptoms with autism.
FDA officials stated they considered approving leucovorin for autism generally but determined there wasn't sufficient data to establish efficacy for autism more broadly. "Right now, we don't have sufficient data to say that we could establish efficacy for autism more broadly," a senior agency official told reporters, adding that off-label prescriptions remain an option for patients to discuss with their physicians.
The approval follows months of heightened interest in the drug after federal health officials suggested last September that leucovorin could have broader applications for children with autism. A study this month found that outpatient prescriptions for leucovorin among children ages 5 to 17 rose 71% in the months following that press conference.
Experts have cautioned that only a handful of small studies have been conducted on leucovorin as an autism treatment. The American Academy of Pediatrics cited limited evidence when it said in October that it does not recommend routine use of leucovorin in kids who are on the spectrum. Since then, one of the largest existing studies looking at leucovorin in children with autism was retracted.
The FDA emphasized that the approval specifically addresses cerebral folate transport deficiency due to the FOLR1 variant. "This action may benefit some individuals with FOLR1-related cerebral folate transport deficiency who have developmental delays with autistic features," the agency stated.