Stealth BioTherapeutics Granted Fast Track Designation for Elamipretide

Stealth BioTherapeutics announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its lead investigational candidate, elamipretide, for the treatment of dry age-related macular degeneration (AMD) with geographic atrophy.

"We are pleased the FDA recognizes elamipretide's potential to treat dry AMD," said Reenie McCarthy, Chief Executive Officer at Stealth. "This regulatory support will help facilitate our efforts to address the severe vision loss that impairs the quality of life of millions of elderly patients living with this disease." AMD is the leading cause of blindness in the elderly and dry AMD currently has no FDA-approved treatments.

In November 2016, Stealth announced the initiation of ReCLAIM, an open-label Phase 1 study to evaluate the safety and tolerability of 24 weeks' treatment with daily subcutaneous injections of elamipretide in patients with dry AMD. In early 2019, Stealth plans to initiate a Phase 2b, randomized, double-masked, placebo-controlled clinical study to evaluate the safety and efficacy of subcutaneous injections of elamipretide in patients with dry AMD with geographic atrophy.

The FDA's Fast Track program facilitates the development and review of drugs to treat serious conditions with unmet medical needs. The designation provides the opportunity for more frequent meetings with the FDA over the course of development and allows for rolling submission of individual sections of a New Drug Application for review. The designation also increases the likelihood of eligibility for priority review and accelerated approval if relevant criteria are met.

Elamipretide has also been granted Fast Track designation for the treatment of primary mitochondrial myopathy, Barth syndrome and Leber's hereditary optic neuropathy, three rare primary mitochondrial diseases.

Age-related macular degeneration (AMD), a progressive eye condition which is the leading cause of blindness in the elderly, affects approximately 10 million people in the U.S. AMD affects the center portion of the retina, called the macula, which is responsible for central vision and color perception. Although there are FDA-approved treatments for wet AMD, which affects approximately 10 percent of those suffering from the disease, there are no approved therapies for dry AMD. Dry AMD with geographic atrophy, an advanced form of dry AMD, is characterized by central blind spots leading to permanent loss of vision. The disease is a major contributor to loss of independence and diminished quality of life in older persons.

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