22 September 2025 - The US FDA today initiated the approval of leucovorin calcium tablets for patients with cerebral folate deficiency, a neurological condition that affects folate (a vitamin essential for brain health) transport into the brain. Individuals with cerebral folate deficiency have been observed to have developmental delays with autistic features (e.g., challenges with social communication, sensory processing, and repetitive behaviours), seizures, and problems with movement and coordination.

The FDA has conducted a systematic analysis of literature published between 2009-2024, including published case reports with patient-level information, as well as mechanistic data, and has determined that the information supports a finding that leucovorin calcium can help individuals suffering from cerebral folate deficiency.

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