Elamipretide Wins FDA Orphan Drug Status, Phase 2 Trial Starts
Stealth BioTherapeutics’ lead candidate elamipretide has been granted orphan drug status by the U.S. Food and Drug Administration (FDA) for the treatment of Friedreich’s ataxia (FA). An orphan drug designation seeks to encourage therapies for rare diseases, through benefits such as seven years of market exclusivity upon approval…