FDA Approves Leucovorin for Autism Amid Investment and Regulatory Clarifications

The U.S. Food and Drug Administration (FDA) has approved leucovorin, a prescription form of folinic acid (vitamin B), for treating children with autism. This approval is based on research suggesting a subset of individuals with autism may have antibodies that disrupt folate transport, a critical nutrient for healthy blood cell function.

The Centers for Medicare & Medicaid Services (CMS) administrator Dr. Mehmet Oz, who reportedly invested in a retailer selling folinic acid supplements, was rumored to financially benefit from this FDA decision. However, the Department of Health and Human Services (HHS) clarified that this approval pertains strictly to prescription leucovorin, distinct from over-the-counter supplements sold by companies like iHerb, in which Oz previously held shares but pledged to divest due to his government role.

The HHS stressed that prescription drugs and retail supplements should not be conflated in discussions about market impact or personal financial gain. Public and social media reactions included concerns about potential conflicts of interest and the efficacy of leucovorin in autism treatment; however, the Autism Science Foundation described current research as preliminary and requiring further study before drawing definitive conclusions.

This case highlights ongoing intersections between regulatory decisions, investment disclosure, and public perception in pharmaceutical approvals tied to emerging therapies in neurodevelopmental conditions.