Rare Disease Report

FDA Grants Fast Track Designation to Mitochondrial Disease Therapy

DECEMBER 19, 2017
Mathew Shanley
Yesterday, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to Stealth Biotherapeutics’ lead drug candidate, elamipretide, for the treatment of Lebers hereditary optic neuropathy (LHON).

Additionally, the potential therapy was granted the same designation for the treatment of 2 other rare mitochondrial diseases; primary mitochondrial myopathy (PMM) and Barth syndrome.

In most cases, LHON, a genetic condition, initially presents with a blurring and clouding of a patient’s vision. It is characterized by loss of visual function resulting from impaired cellular energetics and causes neuropathy of the optic nerve and retinal ganglion cells in the back of the eye, leading to severe loss of visual acuity, or sharpness. It can eventually lead to central vision, which is necessary for detailed tasks such as reading, driving, and facial recognition, and can result in legal blindness of one or both eyes.

At present, it affects an estimated 35,000 people worldwide, primarily young men between the ages of 18 and 30 years old.

"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 press 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."

Results from the Phase 2 ReSIGHT trial, which initiated enrollment at a single trial center in Los Angeles, California in the second quarter of 2016, served as the impetus for the designation. Primary endpoints for safety and tolerability were met, and secondary endpoints – including change in visual function, field and electrical response of retinal ganglion cells, and the measurement of retinal biomarkers by spectral domain optical coherence tomography – were also met.

The study administered twice-daily topical eye drop formulation of elamipretide to enrolled patients with LHON for 52 weeks. Top-line results from the study are expected by mid-2018.

The Fast Track designation will allow for expedited review of the potential therapy. At present, there are no approved therapies for the treatment of LHON in the U.S., however, Raxone (idebenone) was approved by the European Medicines Agency for the indication in September 2015.

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