Elamipretide Granted Orphan Drug Status for Barth Syndrome

Currently, there are no FDA approved treatments for Barth syndrome
Currently, there are no FDA approved treatments for Barth syndrome

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. 

For more information call (617) 600-6888 or visit StealthBT.com.