Drug Development for the Therapy of Mitochondrial Diseases

Summary 

During the last 5 years [1] the number of clinical trials for “Mitochondrial Diseases” has almost quadrupled. The very first drug approved by the FDA in February 2023 was Skyclarys (RTA-408) for the treatment of Friedreich’s ataxia. The European Medicines Agency (EMA) approved in 2015 Raxone (Idebenone) as treatment of visual impairment in patients suffering from Leber’s Hereditary Optic Neuropathyu (LHON) and in 2023 Lumevoq (GS010) as gene therapy for the same condition. Russia approved already in 2012 mitochondrial targeted Visomitin (SKQ1) for the thersapy of dry eye syndrome.

Focusing on new experimental drug entities (New Chemcial Entities, NCEs) Professor Volkmar Weissig will give an update of the state-of-the-art in the field of mitochondrial drug development. He will analyze to what extent growing knowledge over the past two decades about the etiology and pathogenesis of mitochondrial diseases is reflected in the design and development of new experimental drugs. Considering all currently registered clinical trials involving NCEs, He will evaluate how far away we are from a cure of mitochondrial diseases.

Reference :

[1] V. Weissig. Drug Development for the Therapy of Mtochondrial Diseases. Trends in Molecular Medicine 2020, 26, 40-57