Stealth Bio Files US Application For Ultra Rare Genetic Disease Candidate

Stealth BioTherapeutics Corp MITO has submitted an elamipretide marketing application to the FDA for Barth syndrome.
The submission is based on results from the SPIBA-001 Phase 3 Retrospective Natural History Control Trial, which compared data from the TAZPOWER Phase 2/3 trial's open-label portion to match natural history controls.
SPIBA-001 met its primary and most secondary endpoints, demonstrating elamipretide-mediated improvements in exercise tolerance, strength, and cardiac function assessments.
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.
Related content: Benzinga's Full FDA Calendar
Yesterday, the United States Patent and Trademark Office issued patents covering elamipretide for Barth syndrome and Barth syndrome-related cardiomyopathy until February 2034.
Price Action: MITO stock is up 3.42% at $1.21 during the premarket session on the last check Tuesday.