FDA pushes GSK to revive old cancer drug for rare autism-linked disorder

The FDA is urging GSK to resubmit its decades-old drug Wellcovorin (leucovorin) for approval to treat cerebral folate deficiency, a rare condition associated with autism, based on existing literature rather than new clinical trials.

Why it matters: If approved, the move could expand insurance coverage for an off-label treatment already used by doctors and potentially help improve speech in some children with autism. But experts warn that the supporting data is limited, and the regulatory process is bypassing usual trial standards.

Backstory: Wellcovorin was withdrawn from the market in 1999 after going generic. The Trump administration is now reviving it based on retrospective studies showing benefits in language function for some children with autism and folate transport issues. About two-thirds of children in a 48-person trial showed language gains.

Zoom in: Despite the limited evidence, FDA Commissioner Marty Makary claims “hundreds of thousands” may benefit from the revival of this medication. As a result, GSK will submit a supplemental application despite not making a statement about leucovorin´s effect on autism. Leucovorin helps bypass folate metabolism issues and is traditionally used in cancer care to prevent the harmful effects of chemotherapy.

Big picture: This marks a rare case of the FDA initiating a drug approval for a new indication, raising questions about regulatory precedent, scientific rigor, and political influence, especially amid simultaneous controversy over Tylenol and pregnancy safety.