FDA approves narrower use for drug floated for autism
By Peter Sullivan
Published on March 10, 2026.
The US Food and Drug Administration has approved the drug leucovorin for a rare neurological condition, not as a treatment for autism symptoms, as previously suggested. The approval has significantly decreased expectations of a new anti-inflammatory drug. The drug is also being used to treat cerebral folate deficiency, which is estimated to affect less than one in a million people. However, "off-label" prescribing has surged since the announcement of the drug's approval.
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