Drug Development for the Therapy of Mitochondrial Diseases

Abstract

Mitochondrial diseases are a heterogeneous group of inherited or acquired devastating disorders that affect the energy metabolism of the body. Many strategies have been investigated, but currently there is no FDA-approved drug that can alleviate disease symptoms or slow disease progression. This review analyzes 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. All currently registered clinical trials involving new experimental drug entities are reviewed to evaluate how far away we are from the first FDA-approved drug therapy for mitochondrial disease.

Keywords: clinical trials; drug development; experimental drugs; mitochondrial diseases.