FDA approves narrower use for drug floated for autism

The Food and Drug Administration on Tuesday approved using the drug leucovorin for a rare neurological condition — and not as a treatment for autism symptoms, as President Trump's health officials had suggested in September.

Why it matters: The label update dials back expectations that there could be a new autism remedy.

• It also represents a significant narrowing of the potential Trump administration officials had suggested for the little-known form of the vitamin folate.

Driving the news: The FDA has now approved the drug to treat cerebral folate deficiency, a condition that some people with autism also have but that is estimated to affect less than one in a million people.

Between the lines: That is well short of the potential promise for treating autism symptoms Trump administration officials suggested in September.

• FDA Commissioner Marty Makary had suggested then that "hundreds of thousands" of children would benefit from the drug, and Health Secretary Robert F. Kennedy Jr. called it an "exciting therapy that may benefit large numbers of children who suffer from autism."

Yes, but: Though the FDA is not approving leucovorin for autism, "off-label" prescribing has surged following the White House event in September.

• A study published in the Lancet this month found a 71% increase in prescriptions after the White House briefing.
• Leucovorin is an existing drug that has traditionally been used alongside chemotherapy treatment.
• "This action may benefit some individuals with FOLR1- related cerebral folate transport deficiency who have developmental delays with autistic features," Makary said in a statement Tuesday.