The U.S. Food and Drug Administration (FDA) has granted GlaxoSmithKline (GSK) an accelerated pathway to update the label of leucovorin, a decades-old drug, for the treatment of cerebral folate deficiency, a metabolic disorder linked to some autism symptoms. The move allows the agency to bypass lengthy generic drug label updates and clinical trials, potentially delivering a policy win for Health Secretary Robert F. Kennedy Jr. and aligning with the “Make America Healthy Again” initiative. GSK aims to complete the new use application swiftly, after which FDA processing could take as little as four to six months.
While the decision could increase insurance coverage for the drug and offer doctors additional justification for prescribing it, experts caution that evidence supporting its use in autism remains limited. Studies to date involve small patient groups, and most were conducted by the same author. Critics, including Harvard Medical School researchers, described the FDA’s approach as “very atypical,” warning that without robust, replicated trials, the label change may be more symbolic than scientifically validated.
Demand for leucovorin has risen following media coverage and promotion by former President Donald Trump, though many medical professionals continue to restrict its use to clinical trials. Mid-stage trials testing a new liquid formulation of leucovorin for early language impairments in autistic children are ongoing, with initial results expected by December. Experts emphasize that larger, more conclusive studies are necessary to confirm its safety and effectiveness.
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