GSK has withdrawn its application for approval of Wellcovorin, a drug that recently gained renewed attention after suggestions from members of the Trump administration and the Food and Drug Administration (FDA) about its potential to treat autism symptoms, according to an April 10 notice.
The withdrawal comes as public interest in Wellcovorin (leucovorin) increased following discussions about its possible use in treating cerebral folate deficiency (CFD), a rare disorder sometimes linked with developmental delays and autistic features. GSK requested termination of the new drug application because "the drug products were no longer marketed," as stated in a Federal Register notice. A GSK spokesperson said the decision was procedural and not related to safety or efficacy concerns, adding that there were never plans to bring back the drug.
Wellcovorin was first approved in 1983 as a treatment for countering toxic side effects of methotrexate, a chemotherapy agent. The company discontinued it in 1997, but generic versions remain available. The Department of Health and Human Services confirmed that these generics will not be affected by GSK's withdrawal petition. "GSK’s action does not affect the availability of generic leucovorin . . . or the labeling of leucovorin products," said a department spokesperson.
Interest resurfaced last September when the FDA asked GSK to refile an application for Wellcovorin as a potential medicine for CFD. This led to an approval last month allowing its use for CFD caused by certain genetic anomalies. However, during a press call, FDA officials admitted there is insufficient evidence supporting leucovorin's broader approval for autism treatment.
The American Academy of Pediatrics commented that "current evidence is insufficient to support prescribing leucovorin for autism in the absence of CFD." In addition, while FDA Commissioner Marty Makary described it as “an exciting treatment” for autism, what resulted was only an expanded label indication specifically for ultrarare CFD cases.
In related actions last September, the FDA updated labels on acetaminophen-containing products such as Tylenol regarding possible links between prenatal exposure and neurological conditions like autism or ADHD. However, they noted at that time that “a causal relationship has not been established,” citing contrary scientific studies including recent large-scale research showing no link between acetaminophen during pregnancy and autism.
