Stealth BioTherapeutics Receives Orphan Drug Designation from the European Medicines Agency for Elamipretide for the Treatment of Barth Syndrome

Stealth Biotherapeutics Corp (Nasdaq: MITO), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the European Medicines Agency (EMA) has granted orphan drug designation (ODD) for elamipretide for the treatment of Barth syndrome, an ultra-rare genetic condition.

“We are pleased that EMA has recognized, by granting ODD for elamipretide for Barth syndrome, the high unmet need for innovative treatments for this ultra-rare genetic condition,” said Chief Executive Officer Reenie McCarthy. “This marks an important step toward advancing our regulatory initiatives in Europe as we work towards potential ex-U.S. partnerships and approvals.  We are committed to increasing medical and regulatory awareness of this devastating condition, which is believed to contribute to often fatal, infant-onset idiopathic cardiomyopathy globally.”

ODD is granted to investigational therapies intended for the safe and effective treatment of rare diseases with an unmet medical need that affect fewer than 5 in 10,000 people in the European Union. This designation provides companies with certain benefits and incentives including clinical protocol assistance, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralized marketing authorization procedure valid in all EU Member States, and if approved, marketing exclusivity in the EU for 10 years.

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