The U.S. Food and Drug Administration announced Tuesday that it has approved expanded use of leucovorin calcium to treat a rare genetic disorder. File Photo by Jim Lo Scalzo/EPA
March 10 (UPI) — The Food and Drug Administration has approved expanded use of leucovorin calcium tablets for treating cerebral folate deficiency, a rare disorder.
The vitamin, a synthetic form of vitamin B9, has been used to treat toxic side effects of chemotherapy. The FDA approval is based on “a systematic review of the published literature on the topic, including published case reports with patient-level information, as well as mechanistic data,” the agency said in a press release.
Cerebral folate deficiency is a neurological condition that affects folate transport into the brain. People with CFD-FOLR1 often have severe developmental delays, movement disorders, seizures and other serious neurological complications.
According to the National Organization for Rare Disorders, fewer than 20 people have been reported to have cerebral folate deficiency in scientific literature, and the exact prevalence of the disease is unknown.
A few small trials have suggested that the drug could help as an off-label treatment for children with autism, and some families have said their nonverbal children have developed more language skills with the vitamin.
The Trump administration has touted the drug as a potential treatment for autism, though there has not been enough research to back up this claim.
An FDA official told reporters Monday that “we don’t have sufficient data to say that we could establish efficacy for autism more broadly” but said the agency is open to companies studying leucovorin in the autism population, CNBC reported.
The American Academy of Pediatrics said the evidence supporting leucovorin use in children with autism is 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 said in a statement.
“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 in a statement. “This action may benefit some individuals with FOLR1-related cerebral folate transport deficiency who have developmental delays with autistic features.”
FDA officials told reporters Monday that they started with a review of leucovorin as an autism treatment before narrowing its approval to a smaller population with cerebral folate deficiency.
“That was the data where we saw the largest effect sizes,” CNBC reported that one FDA official said on the call. “So we narrowed in on that population, just because we felt like that was the strongest both scientific rationale and also the largest treatment effects that could be used to then overcome some of the limitations in the data sources.”
That official said there can be biases with systematic reviews instead of randomized trials, but said that the treatment effects were so large that they outweighed that concern.
The FDA is encouraging manufacturers of leucovorin to increase production. GSK originally marketed the drug from 1983 until 1997. But it said in September that it has no plans to manufacture the product.
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