Rare Disease Report

Mitochondrial Myopathy Treatment Granted Orphan Drug Designation

OCTOBER 03, 2017
Mathew Shanley
In September, the U.S. Food and Drug Administration granted Stealth BioTherapeutics Orphan Drug Designation for its investigational drug candidate elamipretide, intended for the treatment of patients with primary mitochondrial myopathy (PMM).

In June, Phase 2 data from the MMPOWER-2 Continuation Trial that supports the Phase 3 development of elamipretide in PMM was released at the Mitochondrial Medicine Symposium.

Because muscle and nerve cells have high energy needs, common features of mitochondrial disease include muscular and neurological defects. Genetic diseases caused by defects in the mitochondria that result in prominent muscular problems are referred to as mitochondrial myopathies. The symptoms of mitochondrial disease vary from patient to patient, but in most cases, more than one type of cell, tissue, or organ are affected, leading to fatigue, weakness, and exercise intolerance.

In children with mitochondrial myopathy, vision is also at risk, and developmental delays are common.

"We are thrilled to achieve this key regulatory milestone for the treatment of PMM, a debilitating condition characterized by muscle weakness and fatigue with no FDA-approved treatments," said Reenie McCarthy, Stealth's chief executive officer in a press release. "We will continue to work closely with the FDA as we advance into our Phase 3 trial of elamipretide in patients with PMM."

Stealth BioTherapeutics is privately held and focused on the development of therapeutics for diseases involving mitochondrial dysfunction. Results from the clinical-stage biotechnology company’s Phase 2 proved the clinical benefit of treatment for PMM with elamipretide.

The 30 patients enrolled in MMPOWER-2 previously completed MMPOWER, the company’s first clinical trial in the PMM patient population. Findings in the second trial were consistent with findings found in the first. Both trials were instrumental in identifying additional endpoints for the upcoming Phase 3.

Stealth is currently recruiting for the RePOWER trial, an observational study of patients with PMM, and patients enrolled may have the opportunity to participate in the Phase 3 trial to further evaluate the potential efficacy, safety and tolerability of elamipretide for patients with PMM.

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