Stealth Clinches Orphan Drug Designation in DMD

Stealth BioTherapeutics has received Orphan Drug Designation from the U.S. Food and Drug Administration for elamipretide. The candidate received this designation for its potential to treat patients with Duchenne Muscular Dystrophy (DMD), a rare neurologic disease that affects muscles. In addition, the FDA’s Division of Neurology has agreed to a pre-IND meeting to evaluate the path to regulatory approval of elamipretide.

DMD is typically diagnosed primarily in males between the ages of two and eleven years old, according to the National Institutes of Health’s Genetic and Rare Disease Information Center. Early signs of DMD include difficulty sitting, standing, walking and learning how to speak. As the disease progresses, muscles begin to weaken and degrade, leading to atrophy in skeletal and heart muscles. Patients often die in early adulthood, once respiratory or cardiac issues become too severe for treatment. The disease is hereditary, passed through an X-linked recessive chromosome pattern.

To combat the progression of DMD, elamipretide’s peptide compound composition allows it to penetrate the cellular membrane to bind to cardiolipin. After binding, elamipretide induces an increase in mitochondrial respiration and ATP production. Increased ATP production interrupts and potentially reverses oxidative stress. Oxidative stress is a factor in DMD, along with various other dry age-related macular degeneration and primary mitochondrial diseases.

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