MITO - Stealth Bio's elamipretide gets EMA orphan drug status for muscle disorder

European Medicines Agency (EMA) granted orphan drug designation to Stealth BioTherapeutics' (NASDAQ:MITO) elamipretide to treat myopathic mitochondrial DNA depletion syndrome (M-MDS). The company said the designation applies to patients with primary mitochondrial myopathy due to nuclear DNA mutations (nPMM) who are eligible for enrollment in a phase 3 trial called NuPOWER. The trial is enrolling patients at sites in the U.S. and Europe. MDS is a disorder which causes a drop in mitochondrial DNA in affected tissues. M-MDS is caused by mutations in nuclear DNA genes encoding for enzymes responsible for maintenance and replication of mitochondrial DNA. M-MDS is characterized by muscle weakness, and exercise intolerance, among other things. The EMA grants orphan drug status to therapies intended to treat diseases affecting fewer than 5 in 10K people in the EU. MITO +3.72% to $0.22 premarket May 31

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Stealth Bio's elamipretide gets EMA orphan drug status for muscle disorder