FDA approves Leuconorvin only to treat ultrarare brain disorder, rejecting claims
On Tuesday the U.S. Food and Drug Administration approved a label update allowing leucovorin for an extremely rare subset of cerebral folate deficiency but explicitly declined endorsement as treatment or prevention measure used by thousands with autism spectrum disorders . The agency walked back earlier statements made during September White House briefings in which officials touted decades-old drug showed great promise to help hundreds of children who suffer from Autism.
Key Points
-
1The FDA has officially updated labels to approve leucovorin only as a treatment for an ultrarare subset of cerebral folate deficiency, explicitly excluding approval or endorsement for children diagnosed solely with autism.
-
2This regulatory decision marks the first time in five months that federal health officials have walked back previous statements by President Trump and other administration figures who claimed the drug could benefit hundreds of thousands of autistic patients.
Developments
Perspectives
The FDA has updated labels to approve decades-old medication Leuconorvin, but only as a treatment of rare brain disorders resembling Autism Spectrum Disorder (ASD), not ASD itself.
— [Mar 10, 23:45] CNNFDA officials have clarified that the drug is approved for ultrarare cerebral folate deficiency and lacks evidence to treat autism or its related conditions in large numbers of children as previously suggested by health authorities during a White House briefing.
— [Mar 10, 23:45] StatnewsThe FDA has withdrawn support from Leuconorvin for Autism Spectrum Disorder (ASD) after officials had earlier touted the drug's potential benefits to thousands of children with ASD in September during a White House press conference.
— [Mar 10, 23:45] KrdoThe FDA has approved Leuconorvin for ultrarare cerebral folate deficiency without clinical trials and is walking back statements by President Donald Trump that the drug showed great promise to help people with ASD or related conditions.
— [Mar 10, 23:45] KstpThe FDA has approved Leuconorvin for a rare brain disorder resembling autism but explicitly declined to endorse it as an effective treatment specifically targeting Autism Spectrum Disorder (ASD) after earlier claims suggested broad benefits.
— [Mar 10, 23:45] NBC ChicagoThe FDA approved leucovorin as a treatment only for cerebral folate deficiency with receptor 1 gene mutations (affecting approximately one person per million), not autism. This approval contradicts earlier statements by officials who claimed the drug could benefit thousands of children on the autistic spectrum, leading to increased prescription demand and supply shortages despite fewer than 50 cases worldwide being identified globally for this specific condition.
The U.S. FDA approved high-dose vitamin B (leucovorin) for a rare genetic condition affecting approximately one in every million people worldwide and has identified fewer than fifty cases globally. This approval contradicts the September White House briefing where officials promoted leucovar as an effective treatment potential to thousands of children with autism, despite no evidence supporting such claims at that time or since then (the text cuts off before providing further details).