The FDA on Tuesday announced it was approving Wellcovorin, a prescribed, synthetic form of vitamin B9 also called leucovorin calcium, for cerebral folate deficiency in children and adults with a confirmed variant in the folate receptor 1 gene (CFD-FOLR1).
According to the National Organization for Rare Disorders, there were less than 20 individuals who had been documented in medical literature with the disease in 2019.
In September, the FDA said it had initiated the process of making leucovorin available for treating symptoms of autism, at the same time President Trump told pregnant women to "fight like hell" to not take Tylenol on the claim that it could be linked to autism, despite there being no new evidence of that.
"Hundreds of thousands of kids, in my opinion, will benefit. One study found that with kids with autism and chronic folate deficiency, two-thirds of kids with autism symptoms had improvement and some marked improvement," FDA Commissioner Marty Makary said in September.
Children with CFD often present with symptoms that resemble those of autism spectrum disorder due to their brains not receiving enough folate. With how rare the condition is, however, the majority of autistic children do not have this condition.
The American Academy of Pediatrics has previously said that evidence supporting leucovorin use in autistic children is "currently limited," a consensus that senior administration staffers echoed.
"Right now, we don't have sufficient data to say that we could establish efficacy for autism more broadly," an official noted during a press briefing, adding that off-label use might be an option. "It'll be up to patients to talk with their physicians to see if that might be right for them."
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