The Food and Drug Administration on Tuesday approved a drug to treat a rare brain disorder that resembles autism, while pulling back from previous statements made by top health officials that “hundreds of thousands” of children with autism could benefit from taking the drug.
The medication, leucovorin, will be available for children and adults with cerebral folate deficiency, which limits the delivery of folate, a kind of vitamin B, to the brain. The approval was given to GSK, the original manufacturer of Wellcovorin — a branded version of leucovorin — before the company discontinued its production in 1997. The company does not intend to manufacture or market Wellcovorin again.
The approval is welcome news for the roughly one in a million Americans with the rare genetic condition. But the action also signals the administration’s retreat from the September press conference where President Trump and others first suggested leucovorin held great promise as an autism treatment. Senior FDA officials told the AP that the agency narrowed its review of the medication to focus on uses backed by more robust evidence.
Cerebral folate deficiency is not autism, but the two conditions are both characterized by similar developmental delays. Folinic acid, a dietary supplement, has improved symptoms in people with folate deficiency. Some parents of autistic children have turned to leucovorin, which shares the same ingredient, in a bid to boost their kids’ ability to communicate. Results from a handful of studies were middling — and then the largest study demonstrating its efficacy was retracted in January.
The scientific evidence hasn’t stopped Americans from clamoring for the drug. Outpatient prescriptions of leucovorin rose by 71% in the weeks following the press conference, according to a Lancet study published last week.
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