Potential Drug for Primary Mitochondrial Myopathy Granted FDA Fast-Track Status

The U.S. Food and Drug Administration (FDA) has granted Boston-based Stealth BioTherapeutics Fast Track designation for its MTP-131 (also known as Bendavia) for the treatment of primary mitochondrial myopathy.

Genetic mitochondrial diseases are a group of rare inherited disorders generally characterized by systemic mitochondrial dysfunction, impairing patients’ health and well-being. It robs them of their energy and often makes their muscles very weak.

Bendavia is an investigational drug that targets the inner mitochondrial membrane. It can potentially modify the disease by preserving mitochondrial energy and restoring healthy physiology, while decreasing oxidative stress.

The novel drug is currently being studied for the treatment of primary mitochondrial myopathy in the MMPOWER clinical trial. Results are expected midyear this year.

The FDA’s Fast Track program is a process designed to help the development flow and expedite the review of drugs to treat serious conditions in order to fill unmet medical needs. The purpose is to get important new drugs to patients as soon as possible.

In order to fill an unmet medical need, a therapy must:

• Provide a unique response where none existed previously, or
• Provide evidence of the therapy’s superiority compared to existing available therapies, or
• Show better effectiveness or improved effect on serious outcomes, or
• Avoid serious side effects of an available therapy, or
• Improve the diagnosis of a serious condition where early diagnosis leads to improved outcomes, or
• Decrease the toxicity of an available therapy that is common and causes discontinuation of treatment, or
• Show the ability to address emerging or anticipated public health needs.

Furthermore, a drug that has been granted Fast Track status might be eligible to access Rolling Review status, which allows a company to submit completed sections of its biologic license application (BLA) or new drug application (NDA); and also access accelerated approval and priority review.

“There are currently no approved therapies for primary mitochondrial myopathy,” Stealth BioTherapeutics President Reenie McCarthy said in a press release. “The muscle weakness and extraordinary fatigue experienced by these patients make simple daily tasks very challenging. The Fast Track designation acknowledges an understanding of the significant challenges faced by this patient population, and we look forward to working closely with the FDA as we continue to study MTP-131 as a potential therapy.”