Satellos Bioscience, a Canadian drug discovery company developing small molecule therapeutics to regenerate muscle as a new approach to treating muscle diseases and disorders, has received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for SAT-3153, a potential treatment of Duchenne muscular dystrophy.
The FDA grants Orphan Drug Designation to support the development of medicines for underserved patient populations or rare disorders that affect fewer than 200,000 people in the U.S.
The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States.
“Receiving orphan drug designation and rare pediatric disease designations are important milestones in advancing our novel small molecule therapeutic approach to treating Duchenne.
We continue to make important progress in our drug development program and IND-enabling studies, and we are focused on advancing SAT-3153 into the clinic next year.”Frank Gleeson, Chief Executive Officer for Satellos
SAT-3153 is a small molecule designed by Satellos to inhibit a particular kinase protein which the company believes controls Notch polarity within muscle stem cells. The Company’s scientists have previously shown that muscle stem cell polarity is compromised in Duchenne, leading to a deficit in the body’s innate ability to regenerate muscle.
Satellos believes this finding explains the progressive muscle destruction which characterizes Duchenne as the muscle tissue cannot repair itself as intended. The Company’s scientists have shown in preclinical studies that inhibiting this kinase protein target enables the modulation of polarity and muscle stem cell divisions, enhances muscle regeneration, and increases muscle mass and, critically, muscle function.
Defeat Duchenne Canada has proudly funded the Co-Founder and Chief Scientific Officer of Satellos Bioscience, Dr. Michael Rudnicki, to further his research and discovery of a protein called Wnt7a, its role in stem cell division and, potentially, as a treatment for Duchenne. We have also provided a research partnership and infrastructure grant to support the development of Satellos’ novel approach to treating Duchenne.