IRCM Breakthrough Could Lead to a Drug for a Rare Disease

Institut de recherches cliniques de Montréal (IRCM) reported that A drug candidate discovered by Peter W. Schiller, Director of the Chemical Biology and Peptide Research unit at IRCM and Research Professor in the Department of Pharmacology of the Université de Montréal, has been granted Fast Track designation by the Food and Drug Administration (FDA) for the treatment of primary mitochondrial myopathy. The candidate, elamipretide (formerly known as Bendavia), is being developed by Stealth BioTherapeutics Inc.
According to the press release:

Primary mitochondrial myopathy is a type of rare genetic disorder that impairs patient health and well-being. The muscle weakness and extraordinary fatigue experienced by people suffering from this disease make simple daily tasks very challenging. There is currently no FDA-approved treatment for these patients. Peter Schiller, in collaboration with Professor Hazel Szeto, of Cornell University, discovered a family of molecules, among which is elamipretide.

Peter Schiller commented: 

Due to this property, elamipretide could become the first treatment designed for primary mitochondrial myopathy. The FDA’s grant of a Fast Track designation for this treatment is therefore excellent news.

Click here to view the full press release.