Federal regulators on April 10 said they withdrew approval of a drug made by GlaxoSmithKline that officials had said may be able to treat many cases of autism spectrum disorder.
The Food and Drug Administration said in a regulatory notice that it was withdrawing approval for GSK’s Wellcovorin (leucovorin calcium) tablets.
GSK told officials that the drug was no longer marketed and requested the withdrawal, according to the notice.
“Because Wellcovorin is no longer marketed or manufactured by GSK, the [new drug application] has been withdrawn,” a spokesperson for GSK, which has not marketed Wellcovorin since 1997, told The Epoch Times in an email.
Leucovorin, or folinic acid, has long been available in generic form. The withdrawal “does not affect the availability of generic leucovorin for patients with cerebral folate deficiency, or the labeling of leucovorin products,” a spokesperson for the FDA told The Epoch Times in an email.
Officials said in 2025 that the drug appeared to treat autism symptoms.
“We are going to approve a drug called leucovorin for the treatment of autism, and it may help 50 to 60 percent of kids with autism,” FDA Commissioner Dr. Marty Makary said on C-SPAN.
Health Secretary Robert F. Kennedy Jr. said at a press conference that leucovorin was an “exciting therapy that may benefit large numbers of children who suffer from autism.”
“Right now, we don’t have sufficient data to say that we could establish efficacy for autism more broadly,” an FDA official told reporters on a call in March.
Dr. David Danish, a psychiatrist and president of Philadelphia Integrative Psychiatry, who prescribes leucovorin to some of his patients, told The Epoch Times via email that “the science is clear.”
“Many individuals with autism have specific metabolic roadblocks that prevent regular, everyday vitamins from reaching their brain, which heavily impacts speech, mood, and social connection,” Danish said. “Leucovorin acts as a bridge to bypass that roadblock.”
He added, “In my professional opinion, doctors and patients, should not allow this FDA paperwork update to distract from the very real, evidence-based relief this treatment provides to families every single day.”
Some others, including the Autism Science Foundation, have said the evidence base for leucovorin is too small to say that it helps treat symptoms of autism.
“More studies are necessary before a definitive conclusion can be reached,” the foundation says on its website.