The US FDA has been working in collaboration with GSK towards developing treatment to manage symptoms of patients struggling with autism, thereby on 22nd of September the FDA granted approval for Wellcovorin (leucovorin calcium tablets) for patients with Cerebral folate deficiency (CFD).
Cerebral folate deficiency (CFD) is a neurological disorder that impairs the transport of folate-a vitamin vital for brain health-into the brain. Patients with CFD often experience developmental delays with autistic features, such as difficulties in social communication, sensory processing issues, and repetitive behaviors. The condition may also cause seizures, as well as movement and coordination problems.
Furthermore, “The FDA is working with GSK to expand the current Wellcovorin label,” said George Tidmarsh, Director of the FDA’s Center for Drug Evaluation and Research. “This effort reflects the FDA’s commitment to identify opportunities to repurpose drugs to treat chronic diseases. The FDA remains committed to finding and treating the root causes of autism.”
CFD has also been observed in patients with neuropsychiatric symptoms, including autistic features, and the presence of folate receptor alpha autoantibodies. However, data on the use of leucovorin in this group remain limited, and further studies are needed to confirm its safety and effectiveness.
This collaboration marks an important step toward expanding treatment options for patients living with rare neurological conditions.
Learn more: FDA Takes Action to Make a Treatment Available for Autism Symptoms | FDA
