Stealth BioTherapeutics announced that their lead candidate, elamipretide, for the treatment of Leber’s hereditary optic neuropathy (LHON), has gained Fast Track designation from the Food and Drug Administration (FDA).

Elamipretide has also been granted Fast Track designation for treating primary mitochondrial myopathy (PMM) – which gained Orphan Drug Designation in September – and Barth syndrome. A Phase 2, prospective, double-masked, vehicle-controlled study (‘ReSIGHT’) was initiated in June of this year with top-line results expected mid-2018. The trial is investigating the safety and efficacy of elamipretide, delivered as a topical eye drop in patients with LHON. 

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“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,” said Reenie McCarthy, CEO of Stealth. 

A total of approximately 35,000 people worldwide are affected by LHON, which causes central vision loss and can lead to blindness. LHON primarily affects men aged between 18 and 30 years old. 

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