Posted by Michael Wonder on 10 Mar 2026
FDA approves first treatment for patients with cerebral folate transport deficiency
10 March 2026 - The US FDA today approved expanded use of Wellcovorin (leucovorin calcium) tablets for the treatment of cerebral folate deficiency in adult and paediatric patients who have a confirmed variant in the folate receptor 1 gene.
This action reflects the agency’s commitment to accelerating cures and expanding treatment options including for patients with serious and unmet needs. Leucovorin is the first treatment for the rare genetic condition of cerebral folate deficiency.
