FDA Takes Bold Steps: Reviving Old Drug for Autism and Issuing Acetaminophen Warning

The U.S. Food and Drug Administration (FDA) has made two significant announcements that could have far-reaching implications for the pharmaceutical industry and public health. The agency is seeking to bring back a decades-old drug as a potential treatment for autism symptoms while also initiating a label update for acetaminophen products due to potential risks during pregnancy.

Wellcovorin's Potential Revival for Autism Treatment

In an unprecedented move, the FDA has initiated the reapproval process for GlaxoSmithKline's (GSK) Wellcovorin, a drug that has been off the market for 25 years. The regulator is exploring its potential as a treatment for cerebral folate deficiency (CFD), a condition linked to developmental delays with autistic features, seizures, and motor problems.

Wellcovorin, a branded version of leucovorin, was originally approved in 1983 to counteract the toxic effects of chemotherapy drug methotrexate and overdoses from folic acid antagonists. GSK discontinued marketing the drug in 1997, but its active ingredient, leucovorin, remains available as a generic in the U.S.

George Tidmarsh, director of the FDA's Center for Drug Evaluation and Research, stated that the agency is working with GSK to "broaden" Wellcovorin's label. The pharmaceutical company has expressed intent to file a supplemental application for the drug's use in CFD.

The FDA's decision is based on a systematic analysis of studies from 2009 to 2024, including patient-level information and mechanistic data. However, the Department of Health and Human Services cautioned that leucovorin "is not a cure for ASD [autism spectrum disorder] and may only lead to improvements in speech-related deficits for a subset of children."

Acetaminophen Warning for Pregnant Women

In a parallel development, the FDA has initiated a label update for Tylenol and other acetaminophen products. The new labels will reflect evidence suggesting that use during pregnancy could be linked to an increased risk of neurological conditions such as autism and ADHD in children.

The agency cited the Nurses' Health Study II and the Boston Birth Cohort as sources finding this association. However, the FDA emphasized that a causal relationship has not been established and acknowledged the existence of contrary studies in scientific literature.

Despite the lack of conclusive evidence, the FDA has issued a letter to warn physicians nationwide about the potential link between acetaminophen use in pregnancy and adverse neurological outcomes in children. This action comes despite a 2024 study of 2.5 million children born between 1995 and 2019 that found no association between the use of the painkiller and autism.

These developments coincide with Health Secretary Robert F. Kennedy Jr.'s approaching self-imposed deadline to identify the root cause of autism by September, following his April promise to dedicate the agency's resources to a "massive testing and research effort."