Stealth BioTherapeutics announced that the FDA has granted Fast Track designation for its lead candidate, elamipretide, for the treatment of Leber's hereditary optic neuropathy (LHON).
"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 in a company news release. "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."
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. The disease, which affects approximately 35,000 people worldwide, can lead to legal blindness.
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. Topline results from the study are expected by mid-2018.
Elamipretide has also been granted Fast Track designation for the treatment of primary mitochondrial myopathy (PMM) and Barth syndrome, two rare primary mitochondrial diseases.
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 a drug company to submit individual sections of its New Drug Application (NDA) for review as they are ready, rather than waiting until all sections of the NDA are complete. The designation also increases the likelihood of eligibility for priority review and accelerated approval if relevant criteria are met.