FDA Approves Leucovorin Calcium Tablets to Treat Cerebral Folate Deficiency

The FDA announced the approval of leucovorin calcium tablets to treat cerebral folate deficiency (CFD) following a systematic analysis of literature published between 2009 and 2024. The FDA will partner with GlaxoSmithKline (GSK) to relabel the drug for adult and pediatric patients with CFD.

“This effort reflects the FDA’s commitment to identify opportunities to repurpose drugs to treat chronic diseases,” George Tidmarsh, MD, PhD, director of the FDA’s Center for Drug Evaluation and Research said in an FDA press release.

CFD is a neurological condition that impacts the transportation of folate to the brain. The condition can cause developmental delays, such as challenges with social communication, sensory processing, and repetitive behaviors, as well as seizures and coordination difficulties.

While current patient-level information and mechanistic data indicate the drug has promise in treating this population, the FDA notes that limitations in available data warrant further research to assess safety and efficacy.

Reference:
FDA Takes Action to Make Treatment Available for Autism Symptoms. https://www.fda.gov/news-events/press-announcements/fda-takes-action-make-treatment-available-autism-symptoms. Published Sept. 22, 2025. Accessed Sept. 23, 2025.