Washington– the Food and Drug Administration On Tuesday, he approved a generic medication for a rare brain disorder, during his return Suggestions from President Donald Trump and other administration officials for whom the drug has shown great promise People with autism.
The agency said it approved the use of leucovorin for children and adults who suffer from a genetic condition that limits the access of folic acid, a form of vitamin B, to the brain. FDA officials estimate that this very rare condition affects less than 1 in every million people in the United States
It’s a big step back from the statements made in White House press conference In September, when Trump and F.D.A Marty McCary She announced that the drug is under review to benefit autistic patients, some of whom suffer from a form of brain vitamin deficiency.
“It may be 20, 40, 50 percent of children have autism,” McCary said at the press conference.
The White House event came after promises from Secretary of Health 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 supports the use of the drug by patients with a rare mutation that affects folate levels in the brain.
FDA officials also noted that one study supporting the use of the drug to treat autism was withdrawn earlier this year.
Leucovorin is a synthetic metabolite of folic acid, which is essential for a healthy pregnancy and is recommended for women before conception and during pregnancy. The current FDA label for the drug covers the use of leucovorin in reducing the side effects of some chemotherapy drugs and treating a rare blood disorder.
Patients with the condition targeted with Tuesday’s approval have movement disorders, seizures and other neurological problems that can resemble symptoms of autism.
But professional medical associations say it’s not clear whether the drug helps people with autism.
The American Academy of Pediatrics does not recommend the routine use of leucovorin for children with autism, including those with a brain condition known as cerebral folate deficiency. The group notes that some trials in this subgroup of patients “suggest a potential benefit,” but the research comes from small-scale studies.
Unresolved questions about the drug did not prevent American doctors from prescribing it.
A Published paper The Lancet found last week that leucovorin prescriptions for children ages 5 to 17 were 71% higher than usual in the three months following Trump’s late September news conference. Families of children with autism have reported difficulty obtaining prescriptions in recent weeks.
FDA officials told reporters that the agency is allowing imports of the drug by foreign drug makers to help boost supply. The original manufacturer of the drug, GSK, does not plan to relaunch its version of the drug.
Trump officials originally decided to review the drug after speaking with… Arizona-based neurologist who prescribes medication to autism patients and runs an online education company focused on experimental therapy.
The theory behind the use of the drug is that some people with autism have specific antibodies that prevent 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.
Although there is no single cause of autism, most researchers say science points to genetic and environmental factors as playing a role.
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