FDA says little evidence that a generic drug helps many people with autism

The FDA approved generic leucovorin for an ultrarare genetic disorder that limits folate delivery to the brain, and said evidence mainly supports use for that specific mutation rather than broadly for people with autism.

The Food and Drug Administration approved a generic drug, leucovorin, for a rare brain disorder that limits delivery of folate to the brain. FDA officials said their review focused on the strongest evidence and found support mainly for patients with that rare genetic mutation. The decision followed public statements in September by the White House and FDA commissioner Marty Makary that suggested broader benefits for people with autism. Senior FDA officials also noted that one study that had supported the drug's use in autism was retracted earlier this year. Key facts: - The approval covers leucovorin for children and adults with a genetic condition that impairs folate delivery to the brain, an ultrarare condition estimated to affect fewer than 1 in a million people in the U.S. - Leucovorin is a metabolite of folate and is already included on the FDA label for reducing certain chemotherapy side effects and treating a rare blood disorder. - FDA officials narrowed their review to the strongest available evidence, which they said supports use primarily for patients with the specific folate-delivery mutation. - The American Academy of Pediatrics does not recommend routine use of leucovorin for autistic children, noting that some small trials suggest possible benefit in a subset but evidence is limited. - A recent paper reported a 71% increase in leucovorin prescriptions for children aged 5 to 17 in the three months after the September news conference, and families have reported difficulties getting prescriptions filled. Summary: The FDA approval clarifies that the drug is intended for a specific, ultrarare cerebral folate deficiency rather than as a broadly supported treatment for autism. Professional societies and officials say evidence for benefit in people with autism remains limited, and a supporting study was retracted. Undetermined at this time.