KIT-13 Receives EMA Orphan Drug Designation for Rett Syndrome
On June 23, 2025, the European Medicines Agency (EMA) granted Orphan Drug Designation (ODD) in the European Union for KIT-13, NeuroCores, Inc.’s lead therapeutic candidate for the treatment of Rett Syndrome.
This recognition follows designations from the US FDA, which granted KIT-13 both ODD and Rare Pediatric Disease Designation (RPDD) in 2023. These regulatory milestones mark an important step forward in the program’s progress and reflect the urgency to develop treatment options for Rett Syndrome.
KIT-13 is an oral small molecule, synthetic plasmalogen derivative, developed based on more than two decades of plasmalogen research biology. As a potential first-in-class therapy, KIT-13 is being developed to address the significant unmet needs in Rett Syndrome, a rare pediatric neurodevelopmental disorder that currently has few therapeutic options.
