FDA approves synthetic vitamin B9 for cerebral folate deficiency but not for autism symptoms

The FDA approved leucovorin (a synthetic form of vitamin B9) to treat cerebral folate deficiency, an ultra-rare neurological condition, and agency officials said there is insufficient evidence to support its use for autism symptoms.

The Food and Drug Administration on Tuesday expanded the approved use of leucovorin, a synthetic form of vitamin B9, to treat cerebral folate deficiency. The decision covers Wellcovorin/leucovorin for patients with confirmed variants in the folate receptor gene in some cases. Agency officials emphasized the approval is for the rare condition and not for treating autism more broadly. Senior officials had earlier promoted the drug as potentially helpful for autism symptoms, which prompted public attention. Key details: - The FDA granted expanded approval of Wellcovorin (leucovorin) for cerebral folate deficiency, including cases linked to FOLR1 gene variants. - A senior FDA official said there is not sufficient data to establish efficacy of leucovorin for autism more broadly. - President Trump and FDA Commissioner Marty Makary publicly promoted the drug in September as potentially benefiting children with autism, remarks that drew scrutiny from autism experts. - Published data show leucovorin prescriptions for children rose about 71% in the 2½ months after the September announcement. - Cerebral folate deficiency is described as ultra-rare; estimates include about one in 1 million people and fewer than 20 documented cases reported in the literature as of 2019. Summary: The approval confirms an expanded label for leucovorin to treat cerebral folate deficiency while officials said the evidence does not support approval for autism symptoms. Undetermined at this time.