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FDA approves leucovorin as first drug for rare genetic disorder, after touting it as autism treatment
The FDA approved leucovorin for less than 1 in 1 million patients with cerebral folate deficiency, rejecting broader autism treatment claims after a systematic literature review.
- On Tuesday, the U.S. Food and Drug Administration approved leucovorin as the first labeled treatment for receptor‑1 cerebral folate deficiency in children and adults.
- Senior FDA officials said the review narrowed to strongest evidence for patients with the folate receptor‑1 mutation and relied on a systematic literature review, not randomized, placebo‑controlled clinical trials.
- Published patient‑level reports documented the ultrarare condition affects fewer than 50 cases worldwide, with children becoming seizure‑free or asymptomatic after leucovorin treatment, case studies show.
- After prescriptions surged, officials said they would permit foreign imports to ease shortages as families of children with autism report difficulty filling leucovorin prescriptions.
- The White House had touted the drug in September, prompting heightened attention, but medical societies remain skeptical, a supporting autism study was retracted earlier this year, and GSK does not plan to relaunch the branded product.
73 Articles
73 Articles
Leucovorin lacks evidence to use as autism treatment, FDA says
The Food and Drug Administration on Tuesday approved a drug to treat a rare brain disorder that resembles autism, while pulling back from previous statements made by top health officials that “hundreds of thousands” of children with autism could benefit from taking the drug. The medication, leucovorin, will be available for children and adults with cerebral folate deficiency, which limits the delivery of folate, a kind of vitamin B, to the brain…
kioncentralcoast.com
noticiasya.comBy Brenda Goodman. The prescription drug leucovorin is getting a label update, but it's not what the U.S. Food and Drug Administration (FDA) suggested during a White House briefing in September, when officials touted the drug as a potential treatment for thousands of children with autism. This Tuesday, the FDA approved the high-dose vitamin B — a treatment long used to counteract the side effects of chemotherapy — for folate receptor gene 1 brai…
The Toronto Star
FDA finds little evidence that a generic drug can help people with autism
The Food and Drug Administration approved a generic medication said it approved leucovorin for children and adults with a genetic condition that limits delivery of folate, an essential form of
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