FDA Formally Approves Leucovorin for Rare Folate‑Transport Disorder, Rejects Autism Use for Now
The FDA formally approved generic leucovorin for children and adults with an ultrarare genetic condition that blocks folate transport to the brain (affecting fewer than 1 in a million), but said there is not yet enough evidence to support its use for autism and narrowed its indication to only that mutation. The decision follows the retraction of a key autism study and a White House‑driven surge in pediatric prescriptions last fall, and comes amid shortages that prompted the FDA to allow imports while original manufacturer GSK does not plan to relaunch its branded version.
📌 Key Facts
- The FDA formally approved leucovorin specifically for children and adults with an ultrarare genetic condition that blocks folate delivery to the brain, a disorder estimated to affect fewer than 1 in a million people in the U.S.
- FDA officials explicitly narrowed their review to the strongest evidence and said the only adequately supported indication is the rare folate‑transport mutation; there is not yet enough evidence to support leucovorin for autism more broadly.
- One of the key studies that had supported leucovorin’s use for autism was retracted earlier in the year, further weakening the evidence base for that indication.
- Pediatric leucovorin prescriptions reportedly surged after a late‑September White House news conference promoting the drug for autism — a Lancet analysis found prescriptions for ages 5–17 rose about 71% in the three months after that event — and top Trump health officials (including CMS Administrator Mehmet Oz) publicly oversold the drug’s autism prospects.
- The drug’s review was elevated after Trump administration officials consulted an Arizona neurologist who prescribes leucovorin for autism and runs an online education business around that experimental use.
- Families of autistic children are having trouble filling leucovorin prescriptions amid increased demand; the FDA is allowing imports from foreign drugmakers to ease shortages, while original manufacturer GSK does not plan to relaunch a branded version.
- Autism research groups such as the Autism Science Foundation expressed skepticism about the ‘‘antibody’’ theory motivating some leucovorin use, noting that non‑autistic relatives often have the same antibodies, which undercuts claims that those antibodies cause autism.
📊 Relevant Data
According to 2022 data from the CDC's Autism and Developmental Disabilities Monitoring Network, the prevalence of autism spectrum disorder among 8-year-old children in the US was 36.6 per 1,000 for Black children, 33.0 for Hispanic children, 38.2 for Asian or Pacific Islander children, 37.5 for American Indian or Alaska Native children, and 31.9 for multiracial children, compared to 27.7 for White children.
Prevalence and Early Identification of Autism Spectrum Disorder and Co-Occurring Developmental Delay Among Children Aged 4 and 8 Years — Autism and Developmental Disabilities Monitoring Network, 12 Sites, United States, 2022 — CDC Morbidity and Mortality Weekly Report
Black, Hispanic, and Indigenous children are more likely to be diagnosed with autism later or only when additional intellectual disabilities are present compared to White children.
Autism Society of America Responds to New CDC Report on Updated Autism Prevalence Rates — Autism Society
Research from UCLA indicates that genetic risk factors contributing to autism spectrum disorder in families of Caucasian descent do not influence ASD in the same way in Black families, prompting targeted studies on gene variants in Americans with African ancestry.
Autism in black and white: NIH grant helps scientist study disorder in African Americans — UCLA Newsroom
📰 Source Timeline (3)
Follow how coverage of this story developed over time
- PBS plays an earlier clip of CMS Administrator Dr. Mehmet Oz declaring that 'the FDA, based on NIH research, is approving prescription leucovorin for treatment of autistic children,' underscoring how top Trump health officials publicly oversold the drug’s autism prospects before today’s narrower FDA action.
- The segment stresses that pediatric leucovorin prescriptions 'reportedly surged among children' after that White House‑boosted promotion last fall.
- It reports that today’s FDA decision explicitly approves leucovorin only for a genetic condition that limits folate delivery to the brain and quotes an FDA official saying there is not yet enough evidence that the drug works more broadly for autism.
- Confirms the formal FDA action: approval of leucovorin specifically for children and adults with an ultrarare genetic condition that blocks folate from reaching the brain, estimated to affect fewer than 1 in a million people in the U.S.
- Details that senior FDA officials explicitly narrowed their review to the strongest evidence and state that the only adequately supported indication is the rare folate‑transport mutation, not autism more broadly.
- Adds that one of the key studies supporting leucovorin’s use for autism was retracted earlier in the year, weakening the evidence base.
- Reports a Lancet analysis showing pediatric leucovorin prescriptions for ages 5–17 were 71% higher than normal in the three months after Trump’s late‑September White House news conference touting the drug for autism.
- Notes that families of autistic children are now having trouble filling leucovorin prescriptions and that FDA is allowing imports from foreign drugmakers to ease shortages, while original manufacturer GSK does not plan to relaunch its branded version.
- Highlights that the drug was elevated for review after Trump officials spoke with an Arizona neurologist who prescribes it for autism and runs an online education business around that experimental use.
- Includes Autism Science Foundation and related groups’ skepticism of the "antibody" theory, noting that non‑autistic relatives often have the same antibodies, undercutting claims that they cause autism.