Trending Now - FDA Approves New Use for Vitamin B9, Denies Autism Drug
WHO:
Dr. Alycia Halladay, Autism Science Foundation
David Mandell, autism expert at the University of Pennsylvania
FDA officials
Marty Makary, FDA commissioner
Robert F. Kennedy Jr., Health Secretary
Food and Drug Administration (FDA)
President Trump
Marty Makary, FDA commissioner
Robert F. Kennedy Jr., Health Secretary
American Academy of Pediatrics
Autism Science Foundation
GSK (GlaxoSmithKline)
WHAT:
Dr. Halladay stated, “There is no evidence to say that leucovorin will help most people with autism, and there’s certainly no evidence to say it’s safe.”
David Mandell noted, “We’ve seen huge increases in leucovorin prescriptions for autism because of the initial premature and ill-informed announcement that it can treat autism symptoms.”
The FDA approved leucovorin for a rare genetic condition that limits folate delivery to the brain, but not for autism.
FDA officials stated that their review was narrowed to focus on the strongest evidence, which only supported the drug's use for patients with the rare mutation.
FDA officials noted that one study supporting the drug's use for autism was retracted earlier in 2026.
Leucovorin is a synthetic metabolite of folate, essential for healthy pregnancies.
The FDA's current label for leucovorin includes its use in reducing side effects of certain chemotherapy drugs and treating a rare blood disorder.
A paper published in The Lancet on March 4th, 2026, found that leucovorin prescriptions for children aged 5 to 17 were 71% higher than normal in the three months immediately following Trump's late September news conference.
The FDA approved leucovorin for children and adults with a rare genetic condition that limits folate delivery to the brain.
The ultrarare condition impacts fewer than 1 in a million people in the U.S.
FDA officials stated that their review was narrowed to focus on the strongest evidence, which only supported the drug's use for patients with the rare mutation.
One study supporting the drug's use for autism was retracted earlier in 2026.
The American Academy of Pediatrics does not recommend routine use of leucovorin for autistic children.
Leucovorin prescriptions for children aged 5 to 17 were 71% higher than normal in the three months following Trump’s late September news conference.
Families of children with autism have reported trouble getting prescriptions filled.
WHERE:
Washington, D.C.
Arizona (where the neurologist who prescribes the drug is based)
WHEN:
The FDA approved leucovorin on March 10th, 2026.
The White House news conference occurred in September 2025.
The retraction of the study occurred earlier in 2026.
WHY:
The FDA's review was narrowed to focus on the strongest evidence, which only supported the drug's use by patients with the rare mutation that impacts folate levels in the brain.
The FDA's decision was influenced by the need to focus on the strongest evidence regarding the drug's efficacy for the rare genetic condition.
The FDA's decision was influenced by the need to focus on the strongest evidence regarding the drug's efficacy.
Trump officials decided to review the drug after discussions with a neurologist who prescribes it for autism patients.
HOW:
FDA officials are allowing imports of the drug by foreign drugmakers to help boost supply.
FDA officials announced they are allowing imports of the drug by foreign drugmakers to help boost supply.
The FDA is allowing imports of the drug by foreign drugmakers to help boost supply.
The theory behind the drug's use is that some people with autism have specific antibodies that block folate from entering the brain.
(UPDATED March 11, 2026 05:20 UTC)
In a recent discussion, Dr. Alycia Halladay from the Autism Science Foundation emphasized that there is no evidence to support the claim that leucovorin is beneficial or safe for most individuals with autism. David Mandell, an autism expert at the University of Pennsylvania, pointed out the significant rise in leucovorin prescriptions due to a premature announcement suggesting it could alleviate autism symptoms. Meanwhile, the FDA has narrowed its review to focus on the strongest evidence, which only supports the drug's use for patients with a rare mutation affecting folate levels in the brain, and is now allowing imports from foreign drugmakers to increase supply.
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(UPDATED March 11, 2026 01:25 UTC)
In a significant development, the FDA has approved leucovorin for a rare genetic condition that affects folate delivery to the brain, but not for autism, as officials emphasized their review focused on the strongest evidence available. FDA Commissioner Marty Makary and Health Secretary Robert F. Kennedy Jr. noted that one study supporting its use for autism was retracted earlier this year, while the current label for leucovorin includes its use in reducing chemotherapy side effects and treating a rare blood disorder. To enhance supply, the FDA is also allowing imports of the drug by foreign manufacturers.
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(UPDATED March 10, 2026 15:14 UTC)
Today, the FDA approved leucovorin for children and adults with a rare genetic condition that affects folate delivery to the brain, a condition impacting fewer than one in a million people in the U.S. This decision comes after a review that focused on the strongest evidence supporting the drug's use, particularly following a retracted study earlier this year, and despite the American Academy of Pediatrics not recommending routine use of leucovorin for autistic children. In the three months following a news conference by Trump in September, prescriptions for leucovorin in children aged 5 to 17 surged by 71%, leading to reports from families struggling to get their prescriptions filled.