Overview
- The March 10 decision expands leucovorin’s label to adults and children with confirmed FOLR1 variants causing cerebral folate transport deficiency, an ultra-rare condition affecting roughly 1 in 1 million people.
- Senior FDA officials said evidence does not establish efficacy for autism, noting limited and inconsistent studies and the recent retraction of a major randomized trial.
- The approval relied on a systematic review of case reports and mechanistic data rather than randomized controlled trials, which the agency said are impractical for such a rare disease with large observed effects.
- The updated indication applies to generic formulations because GSK will not manufacture Wellcovorin; the FDA is allowing imports and urging manufacturers to boost supply, and the agency said it does not consider the drug to be in shortage.
- A Lancet analysis found new pediatric prescriptions rose about 71% after a September White House briefing that promoted broad autism benefits; the American Academy of Pediatrics does not recommend routine use for autistic children, and listed side effects include rash, dyspnea, impaired thermoregulation, and anaphylaxis.