US FDA to expand leucovorin’s approved use, but not for autism, WaPo reports

March 10 (Reuters) – The U.S. Food and Drug Administration will announce on Tuesday it will expand approval for leucovorin, but only for a rare condition that some people with autism also have and not for autism itself, the Washington Post reported, citing officials.

The approval is for cerebral folate deficiency, a genetic condition that can cause autism-like symptoms, the report said.

Leucovorin had been approved by the FDA for counteracting the toxic effects of certain cancer drugs, like methotrexate, that block the body’s use of folate.

The report said FDA officials initially considered broadening leucovorin’s use to more people on the autism spectrum, but a systematic review led them to focus on patients with cerebral folate deficiency where the evidence was strongest and effect sizes were largest.

Last year, the FDA commissioner Marty Makary promoted the decades-old drug from GSK, saying it could help “hundreds of thousands” of children with autism. Pediatricians and specialists caution the science on leucovorin in people with autism is limited and does not support widespread use.

The U.S. Department of Health and Human Services, which oversees the FDA, did not immediately respond to a Reuters request for comment outside regular working hours.

(Reporting by Akanksha Khushi and Puyaan Singh in Bengaluru; Editing by Andrew Heavens and Krishna Chandra Eluri)