On September 22, 2025, the FDA announced that it has initiated the process to approve leucovorin calcium tablets for the treatment of cerebral folate deficiency (CFD), a neurological disorder that impairs the transport of folate to the brain.

CFD has been associated with developmental delays with autistic traits, including challenges with social communication, sensory processing, and repetitive behaviors, as well as seizures and problems with movement and coordination.

The decision is based on a systematic review of scientific literature published between 2009 and 2024 (including case reports with patient-level data and mechanistic evidence), from which the FDA concluded that leucovorin may help people with CFD.

WHOM DOES IT IMPACT?

The direct impact is on adults and pediatric patients diagnosed with CFD, medical professionals, and manufacturers.

CONCLUSION:

This is a significant advance for a clinical niche, accelerates access, and clarifies the use of a drug already available for CFD, with the potential to improve symptoms that may include autistic traits, but it does not represent a paradigm shift for the entire autism spectrum.