By MATTHEW PERRONE
WASHINGTON (AP) — The U.S. Food and Drug Administration on Tuesday approved a generic drug for a rare brain disorder, while distancing itself from suggestions by President Donald Trump and other administration officials that the drug showed great promise for people with autism.
The agency reported approval of leucovorin for children and adults who have a genetic disorder that limits the brain’s supply of folate, a form of the B vitamin. FDA officials estimate that the extremely rare condition affects fewer than 1 in every million people in the United States.
This is a significant departure from statements made in September at a White House press conference, when Trump and FDA Commissioner Marty Makary announced that the drug was under review to benefit patients with autism, some of whom have a form of brain deficiency of the vitamin.
“It could be 20, 40, 50% of children with autism,” Makary said at the press conference.
The event at the White House came after Health Secretary Robert F. Kennedy Jr. promised 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 supports use of the drug in patients with the rare mutation that affects folate levels in the brain.
FDA officials also noted that a study supporting the drug’s use for autism was withdrawn earlier this year.
Leucovorin is a synthetic metabolite of folate, which is essential for maintaining healthy pregnancies and is recommended for women before conception and during pregnancy. The current FDA label covers the use of the drug to reduce the side effects of certain drugs used in chemotherapy and to treat a rare blood disorder.
Patients with the condition targeted by Tuesday’s approval have 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 does not recommend routine use of leucovorin in children with autism, including those with the brain condition known as cerebral folate deficiency. Some trials conducted in that subgroup of patients “suggest a possible benefit,” the organization says, but the research comes from small-scale studies.
Unresolved questions about the drug have not stopped doctors in the United States from prescribing it.
A paper published last week in The Lancet found that leucovorin prescriptions for children ages 5 to 17 were 71% higher than normal in the three months immediately following Trump’s press conference in late September. And families of children with autism have reported difficulty getting prescriptions filled in recent weeks.
FDA officials told reporters that the agency allows imports of the drug by foreign manufacturers to help increase supply. GSK, the original manufacturer of the drug, does not plan to relaunch its version of the drug.
Trump administration officials originally decided to review the drug after speaking with an Arizona-based neurologist who prescribes it to patients with autism and runs an online education business focused on the experimental treatment.
The theory behind using the drug is that some people with autism have specific antibodies that block folate from entering the brain. But the Autism Science Foundation and other groups note that non-autistic relatives of people with the disorder often have the same antibodies, suggesting they are not a factor in the condition.
While there is no single cause behind autism, most researchers say that science shows that genetic and environmental factors play a role.
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The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute’s Science and Education Media Group and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
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This story was translated from English by an AP editor with the help of a generative artificial intelligence tool.
