FDA approves Wellcovorin for ultra-rare disease. The Trump admin touted it as a treatment for autism

The Food and Drug Administration (FDA) on Tuesday granted expanded approval to Wellcovorin for the ultra-rare disease cerebral folate deficiency (CFD) in both children and adults, the same prescription drug that the Trump administration touted from the Oval Office as a potential treatment for symptoms of autism last year.

Senior administration officials announced that Wellcovorin, a prescribed, synthetic form of vitamin B9 also called leucovorin calcium, will be approved for use in treating people with CFD who have 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.

GlaxoSmithKline, the company that manufacturers Wellcovorin, stopped selling leucovorin calcium in 1999 when it became available as a generic.

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.

“This is going to be the first FDA-approved treatment for this ultra rare disease that is manifest with neurological complications, including seizures and developmental delay,” a senior health official said during in a press briefing.

“And the approval is exciting because it was based on a systematic review of the literature,” they added. “It did not involve a randomized control trial, but the neurology review team did an exceptional job with their systematic review and was able to show that this was — had a high level of efficacy compared with what we see in the natural history of the cerebral folate transport deficiency disease.”

In September, the FDA said it had initiated the process of making leucovorin available for treating symptoms of autism. This was announced the same day 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.

“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,” FDA Commissioner Marty Makary said in a statement.

“This action may benefit some individuals with FOLR1- related cerebral folate transport deficiency who have developmental delays with autistic features.

Makary claimed in September that “hundreds of thousands of kids will benefit” from the administration promoting Wellcovorin, pointing to data that the drug helped children with autism symptoms. A senior administration official claimed Makary was talking about “cerebral folate deficiency in general” when he made those remarks.

“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,” Makary said in September.

According to the American Academy of Pediatrics (AAP), the evidence supporting leucovorin use in children with autism is “currently limited.”

“Small studies show benefits to communication and behavior for some autistic children, specifically those with CFD or evidence of folate metabolic differences. Larger independent trials are warranted to better understand which patients may benefit,” the AAP’s guidance stated.

A senior administration official said staffers did look into the available data and considered autism when conducting their systematic review.

“Right now, we don’t have sufficient data to say that we could establish efficacy for autism more broadly,” the official noted, 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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