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FDA approves leucovorin as first drug for rare genetic disorder, after touting it as autism treatment
FDA approved leucovorin for cerebral folate deficiency affecting fewer than 1 in a million, but evidence is insufficient to support its use for autism, experts say.
- 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drug in September, prompting heightened attention, but medical societies remain skeptical, a supporting autism study was retracted earlier this year, and GSK does not plan to relaunch the branded product.
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FDA Reverses Stance on Leucovorin for Autism
(MedPage Today) -- The FDA expanded the approval of leucovorin calcium (Wellcovorin) tablets to treat FOLR1-related cerebral folate transport deficiency (CFD-FOLR1), an ultra-rare hereditary disorder, the agency announced Tuesday. Leucovorin is...
·New York, United States
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Doctor explains FDA's refusal to endorse drug once touted as autism treatment
South Shore Health's Dr. Todd Ellerin said the drug Leucovorin does not offer any benefits for autism treatment. The FDA said there is not enough evidence that the drug would not work nor is it recommended for patients.
·Boston, United States
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Donald Trump announced in September that the "new treatment" gives hope "to many parents of autistic children that there is a possibility that their lives can improve" but it was ultimately not approved.
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Total News Sources106
Leaning Left23Leaning Right8Center64Last UpdatedBias Distribution67% Center
Bias Distribution
- 67% of the sources are Center
67% Center
L 24%
C 67%
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