Stealth BioTherapeutics Corp (NASDAQ: MITO) announced that it had received Orphan Drug Designation for elamipretide from the FDA. This drug treats Friedreich’s ataxia.
The company has initiated a Phase IIa clinical trial for the drug in Friedreich’s ataxia. Dr. David Lynch is leading the project at a children’s hospital in Philadelphia. The study also evaluates the cardiac function, visual function, and efficacy of elamipretide after two doses.
Features of Friedreich’s ataxia
Friedreich’s ataxia, a rare disorder, is a genetic illness with no authorized treatments. About 4099 people in the U.S suffer from the disease. The disease’s cause is a frataxin gene defect that leads to frataxin deficiency. The frataxin deficiency, in turn, causes oxidation stress and excessive accumulation of iron in the mitochondria.
One symptom of Friedreich’s ataxia is the gradual loss of vision. It also causes progressive cardiomyopathy in over 90% of patients with the disease. When the patient becomes an adult, the condition is fatal, thus causing early death. Friedreich’s ataxia is one of the most common causes of mortality of premature mortality.
Elamipretide increases the levels of frataxin in cells of patients with the disease. It has also been shown to improve cardiac and motor function in animal models.
The Orphan Drug Designation is for rare diseases
According to Reenie McCarthy, the CEO of Stealth BioTherapeutics, the FDA granted the company Orphan Drug Designation. It recognized a high unmet need in patients with Friedreich’s ataxia. McCarthy adds that the company is pleased to have Dr. Lynch on its team. At the same time, it works to find therapies for Friedreich’s ataxia which is responsible for a shortened lifespan and visual impairment.
The FDA grants orphan drug designation to provide companies with incentives to develop drugs affecting less than 200,000 of the U.S population. Once the body gives this designation to a company, it gets several benefits, including a seven-year exclusive marketing deal after regulators approve the drug for marketing and distribution.
Stealth BioTherapeutics is a biotechnology company that commercializes, develops, and discovers treatments for diseases affecting mitochondrial function. Mitochondrial dysfunction is more common in organs that require a lot of energy to work, like the brain, heart, neuromuscular system, and eye.
