Stealth BioTherapeutics Corp (NASDAQ:MITO) acquired momentum Wednesday, after the company said it received orphan drug designation from the European Medicines Agency for elamipretide for the treatment of Barth syndrome.
Said CEO Reenie McCarthy, “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.
“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 five 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.
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. Barth syndrome occurs almost exclusively in males and is estimated to affect one in 200,000 to 400,000 individuals worldwide. There are currently no FDA- or EMA-approved therapies for patients with Barth syndrome.
MITO gained 44 cents, or 30.3%, to $1.89.