FDA & Government News

FDA Approves Therapy for Cerebral Folate Deficiency

Decision addresses a rare neurologic disorder linked to folate transport impairment.

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    FDA approved Wellcovorin for cerebral folate deficiency with FOLR1 variants.

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    The condition impairs folate transport to the brain.

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    Symptoms include developmental delays and seizures.

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    Approval backed by systematic reviews and evidence.

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    Significance highlighted by FDA officials for affected patients.

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    Adverse reactions include pruritus and anaphylaxis.

Original Source(s)

FDA Approves Therapy for Cerebral Folate Deficiency

Decision addresses a rare neurologic disorder linked to folate transport impairment.

  • by Kathryn Wighton

  • March 10, 2026

  • 2 min

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