Policy & Regulation
US FDA Grants Stealth BioTherapeutics Orphan Drug Designation for Elamipretide for Treatment of Barth Syndrome
12 April 2018 - - The US Food and Drug Administration Office of Orphan Products Development has granted orphan drug designation to Newton, Massachusetts-based clinical-stage biopharmaceutical company Stealth BioTherapeutics' investigational drug candidate, elamipretide, for the treatment of patients with Barth syndrome, the company said.
Barth syndrome is a rare genetic condition characterized by muscle weakness, cardiac abnormalities often leading to heart failure, recurrent infections, delayed growth, and reduced life expectancy.
In March 2018, Stealth completed enrollment in the TAZPOWER clinical trial, a phase 2/3 crossover study evaluating the effects of daily treatment with elamipretide in 12 patients with genetically confirmed Barth syndrome followed by an open-label treatment extension.
Primary endpoints include change in distance walked during the six-minute walk test and change in total fatigue as measured by a patient-reported outcome measure, the Barth Syndrome Symptom Assessment. Secondary endpoints include additional functional assessments, patient-reported outcomes and safety.
Privately held Stealth is focused on the development of therapeutics for diseases involving mitochondrial dysfunction. Based on encouraging preclinical and early clinical data, the company is investigating its lead product candidate, elamipretide, in three primary mitochondrial diseases: PMM, Barth syndrome, and Leber's hereditary optic neuropathy, as well as in heart failure and dry age-related macular degeneration.


Related Headlines