The Food and Drug Administration approved Tuesday a generic medication for a rare brain disorder, while walking back suggestions by President Trump and other administration officials that the drug showed great promise for people with autism.
The agency said it approved leucovorin for children and adults with a genetic condition that limits delivery of folate, an essential form of vitamin B, to the brain. FDA officials estimate the ultrarare condition impacts fewer than 1 in a million people in the U.S.
It’s a major step back from statements made at a White House news conference in September, when Mr. Trump and FDA Commissioner Marty Makary announced the drug was under review to benefit patients with autism, some of whom have a form of the vitamin brain deficiency.
“It might be 20, 40, 50% of kids with autism,” Dr. Makary said at the news conference.
The White House event followed promises from Health and Human Services Secretary Robert F. Kennedy Jr. to determine the cause of autism by September.
But senior FDA officials told reporters Monday that their review was narrowed to focus on the strongest evidence, which only supported the drug’s use by patients with the rare mutation that impacts folate levels in the brain.
The FDA officials also pointed out that one study supporting the drug’s use for autism was retracted earlier this year.
Leucovorin is a metabolite of folate, which is essential for healthy pregnancies and is recommended for women before conception and during pregnancy. The current FDA label covers leucovorin’s use in reducing side effects of certain chemotherapy drugs and treating a rare blood disorder.
Patients affected by the condition targeted by Tuesday’s approval experience movement disorders, seizures and other neurological problems that can resemble symptoms of autism.
But professional medical societies say it’s far from clear whether the drug helps people with autism.
The American Academy of Pediatrics doesn’t recommend routine use of leucovorin for autistic children, including those with the brain condition known as cerebral folate deficiency. Some trials in that subset of patients “suggest potential benefit,” the group states, but the research comes from small-scale studies.
Unresolved questions about the drug haven’t stopped U.S. doctors from prescribing it.
A paper published in The Lancet last week found that leucovorin prescriptions for children aged 5 to 17 were 71% higher than normal in the three months immediately following Mr. Trump’s late September news conference. And families of children with autism have reported trouble getting prescriptions filled in recent weeks.
FDA officials told reporters the agency is allowing imports of the drug by foreign drugmakers to help boost supply. The drug’s original manufacturer, GSK, does not plan to relaunch its version of the drug.
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