The U.S. Food and Drug Administration (FDA) has taken a significant step forward in approving leucovorin calcium tablets for the treatment of cerebral folate deficiency (CFD) — a rare neurological condition that disrupts the transport of folate, a vitamin essential for brain development and function.
Cerebral folate deficiency has been associated with developmental delays, seizures, coordination problems, and autistic features such as challenges in social communication, sensory issues, and repetitive behaviors.
After reviewing published research from 2009 to 2024 — including case reports and mechanistic studies — the FDA concluded that leucovorin calcium shows meaningful benefit for individuals with CFD.
“We have witnessed a tragic four-fold increase in autism over two decades,” said FDA Commissioner Marty Makary, M.D., M.P.H. “Children are suffering and deserve access to potential treatments that have shown promise. We are using gold standard science and common sense to deliver for the American people.”
The FDA is now working with GlaxoSmithKline (GSK), the manufacturer of Wellcovorin® (leucovorin calcium), to update the official prescribing information so that it includes guidance for use in both children and adults with CFD.
Dr. George Tidmarsh, M.D., Ph.D., Director of the FDA’s Center for Drug Evaluation and Research, emphasized the agency’s broader vision:
“The FDA is collaborating with GSK to broaden the existing Wellcovorin label. This effort reflects our commitment to repurpose existing medicines where possible to treat chronic diseases and, importantly, to address the root causes of autism.”
While CFD is rare, researchers have also identified folate receptor alpha autoantibodies in a wider group of patients with neuropsychiatric symptoms and autistic traits. However, the FDA cautioned that more studies are needed before leucovorin can be formally recommended for this broader population.
This move marks a milestone in the FDA’s efforts to expand treatment options for patients with neurological and developmental disorders — offering new hope for families affected by CFD.
