The US Food and Drug Administration (FDA) has approved leucovorin for the treatment of cerebral folate deficiency (CFD), a rare genetic disorder that can cause developmental delays and autism-like symptoms.

The approval applies to GSK’s branded version Wellcovorin, a form of folinic acid that helps restore folate levels in the brain in patients affected by the condition.

Cerebral folate deficiency is associated with mutations in the FOLR1 gene, which impair the transport of folate into the brain. The disorder can lead to neurological symptoms including communication difficulties, seizures, and motor impairment.

The regulatory decision was based on a review of published medical literature and case reports, reflecting the small number of patients affected by the disorder.

The US FDA clarified that the approval is limited to the treatment of cerebral folate deficiency and does not extend to the broader diagnosis or treatment of autism spectrum disorder.

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