Posted by Michael Wonder on 28 Sep 2020
Khondrion receives rare paediatric disease designation for sonlicromanol from US FDA
28 September 2020 - Designation granted for the treatment of MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) syndrome.
Khondrion today announces that it received a rare paediatric disease designation from the US FDA for sonlicromonal for the treatment of patients with MELAS syndrome.
Sonlicromanol is Khondrion’s wholly-owned, potentially first-in-class oral small molecule and one of the most clinically-advanced disease-modifying drug treatments for mitochondrial disease in development. Currently in Phase 2b clinical development, sonlicromanol has already been granted orphan drug designations for MELAS, Leigh disease and patients with maternally inherited diabetes and deafness (MIDD) syndrome in Europe, and for all inherited mitochondrial respiratory chain disorders in the US.
