A molecule discovered at the Montreal Clinical Research Institute (IRCM) was recently granted two new Fast Track designations by the Food and Drug Administration (FDA) for the treatment of Leber’s hereditary optic neuropathy (LHON) and Barth syndrome, which are both rare genetic diseases. The investigational drug, elamipretide, was first created in the laboratory of Peter W. Schiller, Director of the IRCM’s Chemical Biology and Peptide Research Unit, in collaboration with Professor Hazel Szeto, from Cornell Medical College. Elamipretide is now being developed by the biopharmaceutical company Stealth BioTherapeutics Inc. (Stealth).
LHON is an inherited mitochondrial disease that affects approximately 35,000 people worldwide. It causes the loss of central vision due to damage of the retina’s neurons, which can lead to blindness. The disease usually begins when the patients are between 18 and 30 years old.
Barth syndrome is a rare genetic mitochondrial disease that affects almost exclusively boys. It is characterized by cardiac abnormalities, muscle weakness, recurrent infections, delayed growth and reduced life expectancy. Many children with Barth syndrome die from heart failure or infection during childhood, but patients who reach adulthood can live until their late forties.
The FDA's Fast Track designation supports the review of drugs that could treat serious diseases for which no approved treatment is available. In December 2015 and October 2017, Stealth announced that elamipretide was also granted Fast Track designation and Orphan Drug Designation for the treatment of primary mitochondrial myopathy (PMM), respectively. A phase 2 clinical trial was successfully completed in PMM in 2017, and a phase 3 trial has just begun in a group of approximately 200 patients. Several other trials are ongoing, including a phase 1 trial studying elamipretide in dry age-related macular degeneration.
Source:
Anne-Marie Beauregard, Communication Advisor, IRCM
514 987-5555 | anne-marie.beauregard@ircm.qc.ca
