The Food and Drug Administration on Tuesday approved a drug to treat a rare brain disease similar to autism, but retracted previous statements from top health officials that “hundreds of thousands” of children with autism could benefit from taking the drug.
The drug leucovorin will be available for children and adults with cerebral folate deficiency, which limits the delivery of folic acid, a B vitamin, to the brain. This approval was given to GSK, the original manufacturer of Welcovorin, a branded version of Leucovorin, but the company discontinued production in 1997. The company does not intend to manufacture or sell Welcovorin again.
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The approval is welcome news for the roughly 1 in 1 million Americans with a rare genetic disease. But the move also signals a step back from the administration’s September press conference in which Trump and others first suggested leucovorin held great promise as a treatment for autism. FDA officials told the AP that the agency has narrowed its review of the drug to focus on uses that are backed by more solid evidence.
Cerebral folate deficiency is not autism, but both conditions are characterized by similar developmental delays. The dietary supplement folinic acid has improved symptoms in people with folate deficiency. Some parents of children with autism utilize the same ingredient, leucovorin, to enhance their child’s communication skills. A small number of studies had moderate results, and then the largest study that proved its effectiveness was retracted in January.
Scientific evidence hasn’t stopped Americans from clamoring for this drug. A Lancet study published last week found that outpatient leucovorin prescriptions increased by 71% in the weeks following the press conference.
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