Stealth BioTherapeutics Submits Elamipretide NDA To FDA For Treatment Of Barth Syndrome

(RTTNews) - Stealth BioTherapeutics Corp (MITO) said Tuesday that it has submitted a New Drug Application to the U.S. Food and Drug Administration for its lead product candidate, elamipretide, for the treatment of Barth syndrome.

In Tuesday pre-market trade, MITO was trading at $1.22 up $0.05 or 4.27%.

Barth syndrome is an ultra-rare genetic condition characterized by cardiac abnormalities often leading to heart failure and reduced life expectancy, recurrent infections, muscle weakness and delayed growth. There are currently no FDA- or EMA-approved therapies for patients with Barth syndrome.

The NDA submission is based on results from the SPIBA-001 Phase 3 Retrospective Natural History Control Trial. SPIBA-001 met its primary and most secondary endpoints, demonstrating elamipretide-mediated improvements in assessments of exercise tolerance, strength and cardiac function that are unexpected in the natural course of this progressively debilitating disease.

Previously, elamipretide was granted rare pediatric designation, fast track designation, and orphan drug designation by the FDA, and orphan drug designation by the EMA, for the treatment of Barth Syndrome.