FDA approves leucovorin for cerebral folate deficiency in rare patients
TL;DR Summary
The FDA approved leucovorin (folinic acid) as the first treatment for cerebral folate deficiency—a rare genetic disorder that prevents folate from reaching the brain—in select adults and children. The decision was based on a systematic review of published case data rather than a randomized trial and did not establish broad efficacy for autism, though future autism studies remain possible. The approval covers generic leucovorin and GSK’s Wellcovorin, and regulators urged manufacturers to boost production to meet rising demand; GSK has no plans to relaunch the product.
- FDA approves leucovorin as first drug for rare genetic disorder, after touting it as autism treatment CNBC
- FDA declines to endorse leucovorin for autism, walking back earlier statements suggesting benefit CNN
- FDA approves Wellcovorin for ultra-rare disease. The Trump admin touted it as a treatment for autism The Hill
- FDA approves new use of synthetic vitamin B9 — but not for autism symptoms NBC News
- FDA approves generic drug for rare brain disorder but not autism KOMO
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