Stealth BioTherapeutics announced that the Food and Drug Administration (FDA) has granted Orphan Drug Designation to elamipretide for the treatment of patients with Barth syndrome, a rare genetic disorder characterized by muscle weakness and heart abnormalities, often resulting in heart failure, recurrent infections, delayed growth, and reduced life expectancy.

The TAZPOWER study is a randomized, double-blind, placebo-controlled Phase 2/3 crossover study evaluating the safety and efficacy of elamipretide (daily subcutaneous injections) in 12 male patients (aged ≥12 years) with genetically-confirmed Barth syndrome. The study’s primary endpoints include change in distance walked during the 6 minute walk test and change in total fatigue as measured by the Barth Syndrome Symptom Assessment. The secondary endpoints include additional functional assessments, patient-reported outcomes, and safety. Findings from TAZPOWER will be available later this year.

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Elamipretide was granted Fast Track designation by the FDA in November 2017 for the treatment of Barth syndrome.

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