The FDA’s Fast Track program facilitates the development and review of drugs to treat serious conditions with unmet medical needs. Top-line results from the study are expected by mid-2018.Įlamipretide has also been granted Fast Track designation for the treatment of primary mitochondrial myopathy (PMM) and Barth syndrome, two rare primary mitochondrial diseases. In June 2016, Stealth announced the initiation of ReSIGHT, a Phase 2, prospective, double-masked, vehicle-controlled clinical study to evaluate the safety, tolerability and efficacy of topical eye drop delivery of elamipretide in people with LHON. The disease, which affects approximately 35,000 people worldwide, can lead to legal blindness. LHON is an inherited mitochondrial disease that causes loss of central vision due to damage to neurons in the retina, called retinal ganglion cells, as a result of dysfunctional mitochondria.

“This designation, a first for our ophthalmology program, is evidence of the serious need for new therapies for those suffering from this devastating rare mitochondrial disease.”

“We are pleased with the FDA’s decision to grant Fast Track designation to elamipretide for the potential treatment of LHON,” Stealth BioTherapeutics Chief Executive Officer Reenie McCarthy said. Food and Drug Administration (FDA) has granted Fast Track designation for its lead candidate, elamipretide, for the treatment of Leber’s hereditary optic neuropathy (LHON). BOSTON – Decem– Stealth BioTherapeutics ( Stealth), a clinical-stage biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction, today announced that the U.S.

Fasttrack schedule 2017