F.D.A.'s Approval of a Drug for Autism Upends Review Process [View all]

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Source: New York Times

F.D.A.'s Approval of a Drug for Autism Upends Review Process

The agency expanded use of an old drug as a treatment for some people with autism. The endorsement sent some customers scrambling to buy over-the-counter supplements with the same ingredients.


Dr. Marty Makary, the F.D.A. commissioner, speaking at the White House on Monday. President Trump led a briefing with his top health officials, Health Secretary Robert F. Kennedy Jr. and Dr. Mehmet Oz, the head of the Medicare agency, where he pledged to study the root causes of autism. Tierney L. Cross/The New York Times

By Christina Jewett and Benjamin Mueller
Sept. 23, 2025
Updated 7:09 p.m. ET

In taking the unusual step of approving an old generic drug as a treatment for autism, the Food and Drug Administration stunned some experts by departing sharply from the agency's typical standard for reviewing drugs.

The drug, leucovorin, has long been used to treat the toxic effects of chemotherapy, but it was endorsed as a therapy for some people with autism by President Trump and top U.S. health officials during a White House briefing on Monday.

The move flipped the standard process: Typically, a pharmaceutical company carefully studies a drug, often with input from the F.D.A. on the design of rigorous studies, and then files a formal application for approval.

But in this case, the agency said it reviewed medical research and made the approval decision to expand the drug's use on its own. … "Mr. President, you told us to do what's medically right -- to go bold and not worry about the corporations and the lobbyists," Dr. Marty Makary, the F.D.A. commissioner, said on Monday. "So that's what we're here doing today."

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Reed Abelson contributed reporting.

Christina Jewett covers the Food and Drug Administration, which means keeping a close eye on drugs, medical devices, food safety and tobacco policy.
https://www.nytimes.com/by/christina-jewett

Benjamin Mueller reports on health and medicine. He was previously a U.K. correspondent in London and a police reporter in New York.
https://www.nytimes.com/by/benjamin-mueller

A version of this article appears in print on Sept. 24, 2025, Section A, Page 22 of the New York edition with the headline: F.D.A. Upended Process With Approval of Drug as Treatment for Autism. Order Reprints Today's Paper Subscribe

Read more: https://www.nytimes.com/2025/09/23/health/fda-autism-treatment-leucovorin.html