European Medicines Agency Grants Orphan Drug Designation For Elamipretide

Release Date: 07-Sep-2021

European Medicines Agency (EMA) has allowed orphan drug designation for elamipretide for the treatment of Barth syndrome. Barth syndrome is an extreme-rare genetic condition characterized by cardiac abnormalities which leads to reduced life expectancy, heart failure, recurrent infections, muscle weakness and delayed growth. This disease occurs almost totally in males. It is estimated to affect one in 200,000 to 400,000 individuals in the whole world. Currently, there are no FDA- or EMA-approved therapies for patients suffering from Barth syndrome.


It is given to investigational therapies that have the power to treat rare and life-threatening diseases that affect less than five in 10,000 patients in the European Union. It provides some benefits to companies, like clinical protocol advice, fee reductions, and ten years of protection from competition following regulatory approval.

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