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GSK seeks FDA approval for Leucovorin to treat cerebral folate deficiency and autism symptoms, promising new hope for affected children.
FDA plans on approving a new use for Leucovorin for the treatment of kids with cerebral folate deficiency and autistic symptoms.
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GSK announced on Monday September 22, 2025, that it is submitting a supplemental New Drug Application (sNDA) for Leucovorin (Wellcovorin) to update its label to reflect its new indication for cerebral folate deficiency (CFD). FDA requested GSK to act on the label change as a part of the agency’s plan to investigate opportunities of repurposing older medications as treatment solutions for chronic diseases along with the agency’s planned approval for the treatment option.
“In the coming weeks, the FDA will approve Leucovorin as a treatment for children with cerebral folate deficiency and autistic symptoms. Over half of all American children are insured by Medicaid or CHIP, so upon this FDA label change, states will be required to cover Leucovorin around the country,” the agency leaders said, referencing the federal-state health insurance programs for low-income people.
Agency leaders commissioner Marty Makary, NIH director Jay Bhattacharya, and Centers for Medicare and Medicaid Services Administrator Mehmet Oz contributed to a Politico Magazine opinion piece published September 22, 2025, in which all members directed attention towards research suggesting Leucovorin could aid children with folate deficiencies. The article continues to note evidence suggesting that Leucovorin can assist children diagnosed with autism, helping to improve verbal communications.2 According to an article from Politico, the officials also included a statement in their opinion piece, saying the drug “is not a cure for autism.”2
Scientists have cautioned that current data is limited and that Leucovorin needs more research yet claim the form of vitamin B shows potential in a subset of autism patients.
According to the opinion piece by the agency leaders, observational evidence suggests that mothers who take acetaminophen during pregnancy, especially close to delivery, correlates with subsequent diagnosis of conditions like autism and ADHD in their children.3 The agency leaders continue to mention that family control studies have failed to find correlation between Acetaminophen usage and autism or ADHD diagnoses. Acetaminophen remains the only approved over-the-counter- fever treatment for pregnant women.
The agency leaders continued to touch on Acetaminophen, saying, “In light of this evidence and lack of clear alternatives, we believe acetaminophen should be used judiciously in pregnancy, and under medical supervision of an obstetrician, as a practical, prudent medical approach that balances risks and benefits. This caution could be extended to infants and toddlers.”3
According to ACOG President Steven Fleischman, both the Society for Maternal Fetal Medicine and the American College of Obstetricians and Gynecologists announced it is retaining its guidance on Acetaminophen.
“These studies are looking for associations, they don’t show causation,” said Dr. Peter Bernstein, a member of American College of Obstetricians and Gynecologists clinical consensus committee. “Not treating a fever may increase the risk of the pregnancy, certainly more than the Tyleno(Acetaminophen)l use.”
U.K.’s Medicines and Healthcare products Regulatory Agency chief safety officer also made a statement on Acetaminophen, saying,“Patient safety is our top priority. There is no evidence that taking paracetamol during pregnancy causes autism in children,” MHRA chief safety officer Alison Cave said. “Paracetamol(Acetaminophen) remains the recommended pain relief option for pregnant women when used as directed.”
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