FDA Initiates Unusual Repurposing of Old GSK Drug for Autism Treatment

In an unprecedented move, the U.S. Food and Drug Administration (FDA) has announced plans to approve GSK's long-discontinued drug leucovorin, formerly marketed as Wellcovorin, for the treatment of cerebral folate deficiency associated with autism. This decision, part of the Trump administration's efforts to combat autism, marks a significant departure from traditional drug approval processes.

FDA's Literature-Based Approval Strategy

The FDA's decision to repurpose leucovorin stems from a review of published literature spanning from 2009 to 2024. According to the Health and Human Services Department (HHS), this review suggests that the drug may help alleviate speech-related deficits in some patients with autism. FDA Commissioner Marty Makary expressed optimism about the potential impact, stating, "Hundreds of thousands of kids, in my opinion, will benefit."

Despite the agency's enthusiasm, it acknowledged limitations in the available data and emphasized the need for further studies to fully assess the drug's safety and efficacy in this patient population.

GSK's Response and Regulatory Process

GSK, which developed and sold Wellcovorin from 1983 to 1997, has agreed to submit a supplemental new drug application to include cerebral folate deficiency in the drug's label. The British pharmaceutical company discontinued manufacturing the medicine over two decades ago, and it is now available in generic forms in the U.S.

To facilitate this unusual approval process, the FDA is reactivating GSK's previously withdrawn application and requesting additional paperwork from the company. GSK has stated its intention to incorporate data from FDA-identified case reports of genetically confirmed cerebral folate deficiency into Wellcovorin's prescribing information.

Implications and Controversies

This move by the FDA has raised eyebrows in the pharmaceutical and medical communities due to its departure from standard clinical trial requirements. The agency's reliance on existing literature and case reports, rather than new clinical studies, represents a novel approach to drug approval.

Simultaneously, the Trump administration announced plans to modify the safety label of acetaminophen, citing studies suggesting potential neurodevelopmental effects on unborn babies. This announcement, coupled with President Trump's strong statement advising pregnant women to avoid the drug, has sparked controversy and criticism from medical professionals and lawmakers.

Senator Bill Cassidy, a medical doctor and chairman of the Senate health committee, called for HHS to release new data supporting their claims, emphasizing that current evidence does not support such warnings and expressing concern about leaving pregnant women without pain management options.

As these developments unfold, the pharmaceutical industry and medical community will be closely watching the outcomes and potential precedents set by these unconventional regulatory approaches.