The U.S. Food and Drug Administration (FDA) on Tuesday approved the expanded use of Wellcovorin (leucovorin calcium) tablets.
The approval covers cerebral folate deficiency in adult and pediatric patients who have a confirmed variant in the folate receptor 1 gene (CFD-FOLR1).
Rare Neurological Disease
Cerebral folate deficiency is a neurological condition that affects folate (a vitamin essential for brain health) transport into the brain.
CFD-FOLR1 patients often have autism-like symptoms, including severe developmental delays, movement disorders, seizures, and other serious neurological complications.
Leucovorin is the first treatment for the rare genetic condition of cerebral folate deficiency.
The approval was based on a systematic review of the published literature on the topic, including published case reports with patient-level information, as well as mechanistic data.
FDA Approval Based On Scientific Information
The FDA collaborated with GSK plc (NYSE:GSK), the New Drug Application holder of Wellcovorin, on a process to update the labeling to include the essential scientific information needed for the safe and effective use of the drug for adults and pediatric patients with CFD-FOLR1.
A senior official at the FDA told Reuters the agency initially evaluated the drug under a broad review but ultimately narrowed its focus to the strongest available scientific evidence supporting approval.
In the U.S., leucovorin is indicated to diminish the toxicity and counteract the effects of impaired methotrexate elimination and of inadvertent overdosages of folic acid antagonists.
GSK marketed leucovorin under the brand name Wellcovorin from 1983 to 1997. Leucovorin is now available in the U.S. as a generic drug.
Reuters highlighted that GSK no longer manufactures the drug, but the expanded approval allows generic versions to carry the new indication.
Autism Initiatives
Researchers submitted more than 100 proposals to join the Trump administration’s $50 million Autism Data Science Initiative, with as many as 25 grants slated for award in September 2025.
In October 2025, Texas Attorney General Ken Paxton filed a lawsuit against Johnson & Johnson (NYSE:JNJ) and Kenvue Inc (NYSE:KVUE), accusing the companies of misleading pregnant women about the safety of Tylenol despite evidence suggesting its active ingredient, acetaminophen, may increase the risk of autism and attention disorders in children.
The U.S. Centers for Disease Control and Prevention, in March 2025, reportedly said it is preparing to conduct a large-scale study examining potential links between vaccines and autism despite extensive research that has consistently found no credible evidence connecting vaccines to the condition.
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This article FDA Expands Use Of Generic Drug To Treat Autism-Linked Genetic Disorder originally appeared on Benzinga.com
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