Drug Development for the Therapy of Mitochondrial Diseases
Volkmar Weissig, Midwestern University, College of Pharmacy Glendale, USA
Summary of talk
At present (February 2025), the FDA has approved only one new chemical entity (NCE) as a drug for alleviating symptoms of mitochondrial diseases and slow disease progression. Clinical trials involving many more new agents are underway. In my presentation, I 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 for the therapy of these disorders. I will review all currently registered clinical trials involving NCEs and I will try to evaluate how far away we are from the next FDA-approved drug therapy for mitochondrial disease.
