Home / Health / FDA Approves Rare Disease Drug, Denies Autism Link
FDA Approves Rare Disease Drug, Denies Autism Link
12 Mar
Summary
- FDA approved leucovorin for a rare genetic brain disorder.
- The drug was not approved for treating autism spectrum disorder.
- Some autism researchers state the drug is not proven safe or effective for autism.
The Food and Drug Administration recently approved leucovorin, a generic drug, for a rare genetic condition impacting folate transport to the brain. This ultrarare disorder affects fewer than one in a million people in the U.S. and can cause neurological issues resembling autism symptoms. The approval contrasts sharply with earlier claims by administration officials who suggested the drug might benefit autism patients.
Senior FDA officials clarified that the review focused on the strongest evidence, which exclusively supported the drug's use for patients with a specific mutation affecting brain folate levels. They also noted that a study previously cited in support of its use for autism had been retracted. Medical professionals and organizations, like the Autism Science Foundation, reiterated that leucovorin lacks proven safety and efficacy for most autism cases.
Despite the lack of broad approval, leucovorin prescriptions for children aged 5 to 17 saw a substantial increase following public announcements about its potential autism benefits. The FDA is facilitating imports of the drug to address supply issues, as the original manufacturer does not plan to relaunch its version. Experts caution parents against seeking leucovorin for autism due to potential harm and lack of benefit.
