WASHINGTON (news agencies) — The Food and Drug Administration on Tuesday approved a generic medication for a rare brain disorder, while walking back statements by President Donald 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, a 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 comments made at a White House news conference in September, when 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,” Makary said at the news conference.
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The FDA officials also pointed out that one study supporting the drug’s use for autism was retracted earlier this year.
Autism researchers on Tuesday reiterated that the drug has not been shown safe or effective for the vast majority of people with the brain disorder.
“There is no evidence to say that leucovorin will help most people with autism, and there’s certainly no evidence to say it’s safe,” said Dr. Alycia Halladay of the Autism Science Foundation, in an interview.
Halladay noted there is also no established figure for how many people with autism have a form of the folate brain disorder. Some doctors diagnose the folate condition using a specialty laboratory test that isn’t FDA-approved.
The administration’s White House event touting the drug last year followed promises from Health Secretary Robert F. Kennedy Jr. to determine the cause of autism by September.
Leucovorin is a synthetic metabolite of folate, which is essential for healthy pregnancies and is recommended for women before conception and during pregnancy. The drug’s 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.
Unresolved questions about the drug haven’t stopped U.S. doctors from prescribing it.
