Although FDA Commissioner Marty Makary promised “an exciting treatment” for autism, what the agency delivered was a label expansion for leucovorin to treat the ultrarare cerebral folate deficiency. The regulatory process, which relied on a literature review rather than new evidence, stands in contrast to recent rare disease rejections in which the FDA cited a need for more rigorous evidence.

Original title: “What the Leucovorin Approval Signals—or Doesn’t—About the FDA’s Rare Disease Stance”

The U.S. Food and Drug Administration today approved expanded use of Wellcovorin (leucovorin calcium) tablets for the treatment of cerebral folate deficiency in adult and pediatric patients who have a confirmed variant in the folate receptor 1 gene (CFD-FOLR1).

Original title: “FDA Approves First Treatment for Patients with Cerebral Folate Transport Deficiency ”

The Food and Drug Administration (FDA) on Tuesday granted expanded approval to Wellcovorin for the ultra-rare disease cerebral folate deficiency (CFD) in both children and adults, the same prescription drug that the Trump administration touted from the Oval Office as a potential treatment for ... The Food and Drug Administration (FDA) on Tuesday granted expanded approval to Wellcovorin for the ultra-rare disease cerebral folate deficiency (CFD) in both children and adults, the same prescription drug that the Trump administration touted from the Oval Office as a potential treatment for symptoms of autism last year. The Food and Drug Administration (FDA) on Tuesday granted expanded approval to Wellcovorin for the ultra-rare disease cerebral folate deficiency (CFD) in both children and adults, the same prescrip… “This is going to be the first FDA-approved treatment for this ultra rare disease that is manifest with neurological complications, including seizures and developmental delay,” a senior health official said during in a press briefing. “And the approval is exciting because it was based on a systematic review of the literature,” they added. “It did not involve a randomized control trial, but the neurology review team did an exceptional job with their systematic review and was able to show that this was — had a high level of efficacy compared with what we see in the natural history of the cerebral folate transport deficiency disease.” According to the National Organization for Rare Disorders, there were less than 20 individuals who had been documented in medical literature with the disease in 2019.

Original title: “FDA approves Wellcovorin for ultra-rare disease. The Trump admin touted it as a treatment for autism”