Overview
- Approval covers adults and children with cerebral folate deficiency caused by FOLR1 variants and extends to generic products as well as an updated Wellcovorin label.
- The agency relied on a systematic review of published reports and mechanistic data because randomized trials were not feasible for a condition estimated at roughly 1 in 1 million.
- Senior officials said efficacy for autism could not be established, highlighting the January retraction of one of the largest positive trials.
- Leucovorin prescriptions for children rose about 71% after a September White House briefing that had promoted potential autism benefits, creating access problems.
- To address supply pressures, regulators are urging manufacturers to increase production and are allowing imports, while off-label prescribing for autism remains unsupported by robust data.