Stealth BioTherapeutics’ (NASDAQ:MITO) elamipretide received FDA rare pediatric disease designation for the treatment of Barth syndrome.
Barth syndrome is a very rare genetic condition characterized by cardiac abnormalities that often lead to heart failure, recurrent infections, muscle weakness and delayed growth. There are currently no FDA- or EMA-approved treatments.
“This designation for elamipretide underscores our alignment with the FDA around the significant and urgent unmet medical need for Barth patients,” Reenie McCarthy, Stealth’s CEO, said in a statement.
“We hope to improve the health span of Barth patients, for whom cardiomyopathy, exercise intolerance and debilitating fatigue limit life expectancy and impair quality of life,” she added.
