- The European Medicines Agency (EMA) has granted orphan drug designation to Stealth BioTherapeutics Corp's MITO elamipretide for Barth syndrome, an ultra-rare genetic condition.
- Barth syndrome is characterized by cardiac abnormalities, often leading to heart failure and reduced life expectancy, recurrent infections, muscle weakness, and delayed growth.
- Elamipretide, the company's lead candidate, is a peptide compound that readily penetrates cell membranes and targets the inner mitochondrial membrane where it binds reversibly to cardiolipin.
- Price Action: MITO shares are up 49% at 2.16 during the premarket session on the last check Wednesday.
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
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