Months after Trump administration officials promoted a medication as a possible treatment for autism, the Food and Drug Administration is expanding the drug’s approval, but not for everyone with the developmental disability.
The FDA said Tuesday that it has approved leucovorin for use in adults and children with cerebral folate deficiency who have a variant in the folate receptor 1 gene. It is the first treatment for the rare condition, which shares symptoms with autism.
“Today’s approval represents a significant milestone for patients living with cerebral folate transport deficiency due to the FOLR1 variant, a rare genetic condition that has had no FDA-approved treatment options until today,” said FDA Commissioner Marty Makary. “This action may benefit some individuals with FOLR1-related cerebral folate transport deficiency who have developmental delays with autistic features.”
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During a White House briefing last September, federal health officials suggested that leucovorin, a prescription form of folinic acid, could have broader applications for children with autism.
“Today the FDA is filing a Federal Register notice to change the label on an exciting treatment called prescription leucovorin so that it can be available to children with autism,” Makary said at the time. “Hundreds of thousands of kids, in my opinion, will benefit.”
The news prompted a surge in interest in the drug from families of children with autism. A study out this month found that outpatient prescriptions for leucovorin among children ages 5 to 17 rose 71% in the months following the press conference.
Even at the time, however, official documents about the label change were not quite as grandiose as the statements from the press conference, focusing more specifically on cerebral folate deficiency and noting that individuals with the condition “have been observed to have developmental delays with autistic features.”
Experts 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 Since then, one of the largest existing studies looking at leucovorin in children with autism was retracted.
FDA officials said they did consider approving leucovorin for autism generally.
“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. “It’ll be up to patients to talk with their physicians to see if that might be right for them.”
