On September 25, 2025, the U.S. Food and Drug Administration (FDA) announced the approval of leucovorin calcium tablets for the treatment of cerebral folate deficiency (CFD), a neurological disorder characterized by impaired folate transport into the brain, resulting in developmental delays, autistic features (e.g., social communication challenges, sensory processing issues, repetitive behaviors), seizures, and motor coordination difficulties.
This approval follows a comprehensive FDA analysis of literature from 2009 to 2024, including case reports and mechanistic data, which demonstrated the potential efficacy of leucovorin calcium in alleviating CFD symptoms. The decision addresses a critical need amid a reported four-fold rise in autism diagnoses over the past two decades, emphasizing the urgency to provide effective treatments for affected individuals, particularly children.
The FDA is working with GSK, the New Drug Application holder for Wellcovorin (leucovorin calcium), to revise labeling to include essential scientific information for safe and effective use in both adult and pediatric CFD patients. While CFD has been observed in a broader population with neuropsychiatric symptoms and folate receptor alpha autoantibodies, the FDA notes limitations in current data, necessitating further studies to confirm safety and efficacy in this group.
This approval represents a significant step in repurposing an existing drug to address a root cause of autism-related symptoms, aligning with the FDA’s commitment to innovative solutions for chronic diseases. The agency continues to prioritize rigorous scientific evaluation to ensure safe and effective treatment options, aiming to improve outcomes for individuals with CFD and related autistic features. Additional research is planned to explore the broader applicability of leucovorin calcium and to optimize its use in diverse patient populations, reinforcing the FDA’s dedication to public health and advancing therapies for complex neurological conditions.
