Agency clarifies that the generic drug will be approved only for an ultra-rare genetic disorder linked to cerebral folate deficiency, not as a treatment for autism.
WASHINGTON | The U.S. Food and Drug Administration said this week that current scientific evidence does not support the use of the generic drug leucovorin as a treatment for autism, marking a significant shift from earlier statements by top federal health officials who had suggested the medication could benefit large numbers of children with the condition.
Senior FDA officials told reporters that the agency’s latest decision reflects a commitment to approving therapies only when clear clinical evidence of safety and effectiveness exists. Instead of approving leucovorin for autism-related conditions, regulators authorized the drug for a rare genetic disorder known as FOLR1-related cerebral folate transport deficiency, a condition that affects fewer than one in a million people.
The move effectively narrows the scope of the drug’s regulatory approval after months of public discussion surrounding its potential broader neurological benefits.




















