An IRCM breakthrough leads to a potential drug for a rare disease

Feb. 17 2016 | Rare diseases

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A drug candidate discovered by Peter W. Schiller, Director of the Chemical Biology and Peptide Research unit at the Institut de recherches cliniques de Montréal (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.

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. “Elamipretide targets the inner mitochondrial membrane and helps preserve mitochondrial energetics,” says Peter Schiller. “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.”

The FDA’s Fast Track program facilitates the development and review of drugs to treat serious conditions with unmet medical needs. In addition, the Fast Track program increases the likelihood of eligibility for priority review and accelerated approval if relevant criteria are met. Stealth BioTherapeutics is presently studying elamipretide in rare diseases like mitochondrial myopathy and as a potential therapeutic for cardiovascular and ocular diseases.

About Peter Schiller
Peter W. Schiller holds a PhD in medicinal chemistry and molecular pharmacology from the ETH-Zurich in Switzerland. He is a Full IRCM Research Professor and Director of the Chemical Biology and Peptide Research laboratory.  He is also Full Research Professor in the Department of Pharmacology at the Université de Montréal and member of the Scientific Advisory Board at the Centre for Drug Discovery, at Bouvé College of Health Sciences, Northeastern University, Boston, Massachusetts. Dr. Schiller also holds the Canadian Pacific Chair on Pain. 

About the IRCM
The IRCM (Institut de recherches cliniques de Montréal) is a renowned biomedical research institute located in the heart of Montréal's university district. Founded in 1967, it is currently comprised of 35 research units and four research specialized clinics: nutrition, metabolism and atherosclerosis; hypertension; diabetes and obesity as well as rare diseases such as cystic fibrosis and familial hyperlipidemia. The IRCM employs nearly 425 people. It is affiliated with the Université de Montréal, and the IRCM Clinic is associated to the Centre hospitalier de l'Université de Montréal (CHUM). It also maintains a long-standing association with McGill University. The IRCM is funded by the Quebec ministry of Economy, Science and Innovation (Ministère de l’Économie, de la Science et de l’Innovation). For more information, visit

Additional information:

Source :
Anne-Marie Beauregard, Communications Officer, IRCM
514 987-5555 |

Manon Pepin, Executive Director of Communications and Public Relations, IRCM
514 987-5535 |