Stealth Bio's Elamipretide Gets Orphan Drug Designation From FDA For Friedreich's Ataxia

(RTTNews) - Stealth BioTherapeutics Corp. (MITO) said on Monday that its drug candidate Elamipretide has been granted orphan drug designation by the FDA to treat Friedreich's ataxia.

Friedreich's ataxia, a rare genetic disease affecting an estimated 4,000 individuals in the United States, is caused by a defect in the frataxin gene resulting in a relative deficiency of frataxin, leading to mitochondrial iron accumulation and oxidative stress. This disease is characterized by a progressive decline in visual function and progressive cardiomyopathy.

A phase IIa investigator-initiated trial evaluating two doses of Elamipretide in patients with Friedreich's ataxia to assess safety, visual function, and cardiac function is underway.

Elamipretide is also being studied in a phase II clinical trial in subjects with extra-foveal geographic atrophy associated with dry age-related macular degeneration, dubbed ReCLAIM-2. Topline results from this study are anticipated during the second quarter of 2022.