FDA approves leucovorin as first drug for cerebral folate deficiency

The FDA approved leucovorin (folinic acid) as the first treatment for cerebral folate deficiency after a review of published studies; the decision follows earlier public attention when the drug was discussed as a possible autism therapy.

The Food and Drug Administration approved leucovorin on Tuesday as the first treatment for a rare genetic disorder in certain adults and children. The decision follows months of public attention after the Trump administration highlighted leucovorin as a possible therapy for some autism symptoms, which led to increased prescriptions. The agency said it did not find sufficient data to establish efficacy for autism more broadly and instead focused approval on patients with cerebral folate deficiency. The approval was based on a systematic review of published literature rather than randomized clinical trials. Key details: - Leucovorin, also called folinic acid, is a synthetic form of vitamin B9 that has been used to treat toxic side effects of chemotherapy. - The FDA narrowed approval to patients with cerebral folate deficiency, a rare genetic mutation that prevents folate from properly reaching the brain and can cause severe developmental delays and seizures. - Officials said the strongest data and largest reported treatment effects were found in that specific patient population. - The approval was supported by a systematic review of published studies and case reports, not by randomized controlled trials. - The agency is encouraging manufacturers to increase production to meet higher demand, and GSK has said it does not plan to relaunch the branded product Wellcovorin. Summary: The approval makes leucovorin the first authorized treatment for cerebral folate deficiency and covers both generic formulations and the previously marketed brand. FDA officials said the agency remains open to companies studying leucovorin in broader autism populations but concluded that the best available evidence supported use in the rare genetic disorder. Regulators have asked manufacturers to boost supply to address increased demand.