WASHINGTON (AP) — The U.S. Food and Drug Administration on Tuesday approved a generic drug to treat a rare brain disease, reversing advice from President Donald Trump and other government officials that the drug showed great promise for people with autism.
The agency said it approved folinic acid for use in children and adults with a genetic disorder that limits the delivery of folate, a form of vitamin B, to the brain. FDA officials estimate the extremely rare disease affects less than one in a million people in the U.S.
It’s a major step back from statements made at a White House press briefing in September, when Trump and FDA Commissioner Marty Makary announced that the drug was under review to benefit people with autism, some of whom suffer from a form of vitamin C deficiency in the brain.
“Probably 20, 40, 50 percent of kids are autistic,” Macari told a news conference.
The White House event came after Surgeon General Robert F. Kennedy Jr. pledged in September to identify the cause of autism.
But senior FDA officials told reporters on Monday that their review has been narrowed to focus on the strongest evidence that supports use of the drug only in patients with a rare mutation that affects brain folate levels.
FDA officials also noted that a study supporting the drug’s use in treating autism was withdrawn earlier this year.
Leucovorin, the anabolic form of folic acid, is essential for a healthy pregnancy and is recommended for women to take before conception and during pregnancy. The drug’s current FDA label covers leucovorin’s use in reducing the side effects of certain chemotherapy drugs and treating rare blood disorders.
Patients affected by the conditions targeted by Tuesday’s approval experience movement disorders, seizures and other neurological problems that may resemble symptoms of autism.
But professional medical associations say it’s unclear whether the drug can help people with autism.
The American Academy of Pediatrics does not recommend routine use of leucovorin in children with autism, including those with brain folate deficiency. The team said some trials in this group of patients “suggested potential benefit,” but the research was from a small study.
Unanswered questions about the drug haven’t stopped U.S. doctors from prescribing it.
A paper published in The Lancet last week found that in the three months after Trump’s press conference in late September, leucovorin prescriptions for children aged 5 to 17 were 71% higher than normal. In recent weeks, families of children with autism have reported difficulties getting their prescriptions filled.
FDA officials told reporters the agency is allowing foreign drugmakers to import the drug to help increase supply. The drug’s original manufacturer, GlaxoSmithKline (GSK), does not plan to relaunch a version of the drug.
Trump officials initially decided to review the drug after speaking with a neurologist in Arizona who prescribes the drug to people with autism and runs an online education business focused on experimental treatments.
The theory behind the drug’s use is that some people with autism have specific antibodies that block folic acid from entering the brain. But the Autism Science Foundation and other groups point out 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 science shows genetic and environmental factors play a role.
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