On Sept. 22, 2025, the U.S. Food and Drug Administration (FDA) announced it h initiated the approval of leucovorin calcium tablets for patients with cerebral folate deficiency (CFD), a neurological condition that affects folate (a vitamin essential for brain health) transport into the brain. Individuals with cerebral folate deficiency have been observed to have developmental delays with autistic features (e.g., challenges with social communication, sensory processing, and repetitive behaviors), seizures, and problems with movement and coordination.

The FDA has conducted a systematic analysis of literature published between 2009-2024, including published case reports with patient-level information, as well as mechanistic data, and has determined that the information supports a finding that leucovorin calcium can help individuals suffering from CFD.  

The FDA is working with GSK, the innovator of Wellcovorin (leucovorin calcium), on a process to include the essential scientific information needed for the safe and effective use of these drug products for adults and pediatric patients with CFD. As the New Drug Application (NDA) holder for this medicine, GSK has preliminarily agreed to work with the FDA on this relabeling effort.