As someone with more than 40 years as a regulatory affairs professional in the FDA regulated industry, I’ve earned the right to say:

WHAT THE !@*!*$#!!!!

FDA approved leucovorin for the treatment of autism. Wait just a @*!*$#! minute. GlaxoSmithKline (GSK) marketed the drug they named Wellcovorin (leucovorin calcium.) The drug was approved for the treatment of toxic side effects from chemotherapy.

Basically, FDA decided to re-approve a drug from which they withdrew approval because the manufacturer withdrew the application since they were no longer making it. Yes, I know that’s a confusing sentence. (The Wellcovorin was approved in 1983, FDA withdrew approval 1999 and re-approved in 2025. Nobody makes it.)

“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. “So that’s what we’re here doing today.”

the New York times, 23Sep25

WHAT THE !@*!*$#!!!!

I had to look up the Federal Register Announcement, just to see what FDA really said. Could this be possible?

FDA looked at case studies since 2009. They reviewed reports on 40 people with genetically linked cerebral folate (a vitamin B) deficiencies or CFD. Symptoms of CFD can include “global developmental delays with autistic features and psychomotor regression, intractable epilepsy, and cerebellar ataxia.” Most of the 40 people in the case studies had improved symptoms. Somewhere between 20 and 50% of people with autism have low levels of folate. Just to be absolutely clear, just because someone has genetically linked CFD, does not mean they have autism.

Just a quick side note: Drugs are approved based on controlled clinical studies including many people across different sexes, ages, and ethnicities. Pediatric studies are usually done separately. The studies are designed to test the effectiveness of the drug in treating a specific condition.

Let me get this straight. Based on 40 people with a genetically linked deficiency, FDA is approving a drug? No clinical studies? No application from a pharmaceutical company? That can’t be. Surely, that can’t be.

It isn’t. (Whew!)

FDA is asking GSK to submit an application. The application must include “the essential scientific information needed for the safe and effective use of these drug products for the treatment of CFD in adults and pediatric patients.” That usually means controlled clinical trials.

So, once the application is submitted and the data is reviewed, the FDA may approve leucovorin for the treatment of CFD. Just to hammer this nail one more time, that’s not the same as approving it for the treatment of autism. (You can read the Federal Register announcement here.)

Oh, wait a minute, there’s more.

FDA said on X that leucovorin is “not to be conflated with ANY over-the-counter drugs or supplements.” Nonetheless, supplements containing the active ingredient in GSK’s leucovorin are selling like hotcakes.

A prominent online supplement seller, iHerb, said that it had sold out of its in-house brand of folinic acid on Tuesday. The company’s website said the product would not be back in stock until late November.

the New York times, 23Sep25

Dr. Mehmet Oz, who heads up the Center for Medicare and Medicaid, was a pitchman for iHerb. In fact, he once had holdings in the company valued somewhere $5-25 million. Dr. Oz reported that he had resigned from iHerb and sold his stock.

Seems a little fishy to me. Just saying:

WHAT THE !@*!*$#!!!!