Classification of Evidence
Primary mitochondrial diseases, primary mitochondrial myopathy, fatigue, MMPOWER-1, primary mitochondrial myopathies
MITOCHONDRIAL DISEASES, DISORDERS, METABOLIC DISORDERS
This study provides Class I evidence that elamipretide does not improve the 6MWT or fatigue at 24 weeks compared with placebo in patients with primary mitochondrial myopathy.A consensus of experts define primary mitochondrial myopathies (PMMs) as a diverse group of genetically confirmed disorders of the mitochondria, affecting predominantly, but not exclusively, skeletal muscle, thereby adversely affecting physical function and quality of life.Primary mitochondrial diseases (PMDs) caused by both mitochondrial (mtDNA) and nuclear DNA (nDNA) alteration are among the most common inherited metabolic disorders.Currently, available standards of care primarily use dietary supplements that have limited clinical impact.Elamipretide is an investigational mitochondrial-targeting agent in development for treating patients with a variety of mitochondrial diseases.The elamipretide clinical development program included MMPOWER-1 and MMPOWER-2, whereby treatment with elamipretide demonstrated meaningful improvements in patient-reported outcomes (PROs) for patients with confirmed PMM.