WASHINGTON — Federal health regulators on Tuesday granted approval for a generic medication targeting an extremely rare neurological condition, while simultaneously rejecting earlier White House assertions that the treatment could benefit large numbers of autism patients.
The Food and Drug Administration authorized leucovorin for children and adults diagnosed with a genetic disorder that prevents folate — a crucial B vitamin — from reaching the brain. Agency experts estimate this exceptionally uncommon condition affects less than one person per million Americans.
This decision represents a dramatic departure from claims made during a White House press event in September, where President Donald Trump and FDA Commissioner Marty Makary announced the medication was being evaluated to help autism patients, particularly those with vitamin deficiencies affecting brain function.
“It might be 20, 40, 50% of kids with autism,” Makary stated during that September announcement.
The White House presentation came after Health Secretary Robert F. Kennedy Jr. pledged to identify autism’s underlying causes by September.
However, top FDA officials informed reporters Monday that their evaluation was refined to concentrate on the most compelling scientific evidence, which only validated the drug’s effectiveness for patients with the specific genetic mutation affecting brain folate levels.
The FDA officials also noted that one research study supporting the medication’s autism applications was withdrawn earlier this year.
Leucovorin functions as a folate derivative, which plays a vital role in healthy pregnancies and is advised for women planning conception and during pregnancy. The existing FDA authorization permits leucovorin’s use in minimizing chemotherapy side effects and treating a rare blood condition.
Individuals with the condition addressed by Tuesday’s approval suffer from movement difficulties, seizures, and other neurological complications that may mirror autism symptoms.
Nevertheless, medical professional organizations maintain significant uncertainty about whether the drug benefits autism patients.
The American Academy of Pediatrics does not endorse regular leucovorin treatment for children with autism, even those diagnosed with cerebral folate deficiency. While some clinical trials involving this patient subset “suggest potential benefit,” the organization notes these findings stem from limited research studies.
Outstanding questions regarding the medication haven’t prevented American physicians from prescribing it.
Research published in The Lancet last week revealed that leucovorin prescriptions for children between ages 5 and 17 increased 71% above typical levels during the three months following Trump’s late September announcement. Additionally, families with autistic children have encountered difficulties obtaining filled prescriptions recently.
FDA officials informed reporters the agency is permitting foreign pharmaceutical companies to import the drug to address supply shortages. GSK, the medication’s original producer, has no plans to resume manufacturing its version.
Trump administration officials initially chose to evaluate the drug after consulting with an Arizona neurologist who prescribes it for autism patients and operates an online educational platform promoting this experimental treatment approach.
The underlying theory suggests certain autism patients possess specific antibodies that prevent folate from entering brain tissue. However, the Autism Science Foundation and other organizations highlight that non-autistic family members of people with the disorder frequently carry identical antibodies, indicating they likely don’t contribute to the condition.
Although autism has no singular cause, most researchers agree scientific evidence points to genetic and environmental influences as contributing factors.
