U.S. health officials are quickly taking regulatory action to build on findings from the administration’s research push into the “autism epidemic,” including a label update for a decades-old GSK drug and a safety label change for over-the-counter pain reliever Tylenol (acetaminophen).
Following reports that the White House will look to link Tylenol use during pregnancy to autism, President Donald Trump made his stance clear at a Monday afternoon press conference detailing “significant medical and scientific findings for America’s children.”
“Don’t take Tylenol,” the president repeatedly emphasized, citing a potential association between Tylenol use during pregnancy and a “very increased” risk of autism.
As such, the FDA has already begun the process to change the safety label of acetaminophen to reflect evidence that could suggest an increased risk of autism and other neurological conditions such as attention-deficit/hyperactivity disorder.
The "precautionary principle" of the label edit “may lead many to avoid using acetaminophen during pregnancy, especially since most low-grade fevers don’t require treatment,” FDA Commissioner Martin Makary, M.D., explained in an FDA release.
In the meantime, Makary signed a notice to physicians urging them to “consider minimizing” the use of acetaminophen during pregnancy to treat “routine low-grade fevers.”
The Sept. 22 letter noted that the "consideration should also be balanced with the fact that acetaminophen is the safest over-the-counter alternative in pregnancy among all analgesics and antipyretics" and further clarifies that a causal relationship between acetaminophen use and autism has not been established.
Tylenol had been marketed by Johnson & Johnson since its 1951 FDA approval but recently switched hands to the company’s consumer healthcare spinout Kenvue. Kenvue pushes back on the notion that the drug causes autism, pointing to “independent, sound science,” the company told Fierce Pharma in a statement.
Still, the FDA said it is partnering with acetaminophen makers to update the labeling and drive new research, according to a Department of Health and Human Services (HHS) release. The HHS is set to launch a “nationwide public service campaign” to inform families of the new safety label.
Over in Europe, the EU on Sept. 23 made clear in a statement that its acetaminophen (sold as paracetamol in Europe and other parts of the world) guidelines remain unchanged as the regulator has "found no evidence that taking paracetamol during pregnancy causes autism in children."
'Autism symptom' treatment
Meanwhile, after nailing down Tylenol as a potential cause of autism, the FDA is moving forward with a solution in GSK’s Wellcovorin (leucovorin calcium). Leucovorin has been used since the 1950s to counter the toxicities of certain chemotherapies but is now being positioned as a treatment for the “autistic features” of cerebral folate deficiency.
GSK’s branded version hasn’t been sold since the late 1990s, when the company stopped marketing it and asked that the FDA pull its approval, according to a notice on the Federal Register.
Now, the FDA is reviving the drug. Wellcovorin has been reapproved "on the basis of new data," the notice states, as the agency works to position it as an autism treatment.
To that end, the FDA “initiated the approval” of its use for patients with cerebral folate deficiency, a neurological condition that impacts folate transportation and has been associated with autism. The move is based on a "systemic analysis" of literature published between 2009 and 2024, which supports a finding that the drug can be helpful in cerebral folate deficiency treatment, the agency said.
The treatment would be used for “autistic symptoms” but is not a cure for the condition and may only prompt improvements in “speech-related deficiencies” for a subset of children with autism, the HHS clarified. The National Institutes of Health (NIH) will launch related confirmatory trials and safety studies.
GSK is collaborating with the FDA on the label update, it said in a statement. Although the company no longer makes or markets the now-generic drug, it is submitting a supplemental new drug application at the FDA's request as the original application holder.
The last component of the administration’s autism agenda is a $50 million Autism Data Science Initiative the NIH will spearhead using “large-scale data resources” to weigh contributors to autism prevalence.
The $50 million in awards will support 13 projects exploring a “wide range” of potential environmental, medical and lifestyle factors that could intertwine with genetics or biology, “opening the door to breakthroughs that will deepen our understanding of autism and improve lives,” NIH Director Jay Bhattacharya, M.D., Ph.D., commented.
About 1 in 36 children have been diagnosed with autism spectrum disorder in the U.S., according to the Centers for Disease Control and Prevention, reflecting wider prevalence than the 1 in 150 children reported in 2000. However, advocacy groups such as Autism Speaks have long maintained that greater prevalence of the condition can be chalked up to increased awareness, broader diagnostic criteria and improved screening tools.