In a recent announcement from Sanofi Genzyme, we’ve learned that the U.S. Food and Drug Administration (FDA) has approved Nexviazyme® for the treatment of late-onset Pompe patients that are one year old and older. This decision is good news for the Pompe community in the U.S. Nexviazyme had been granted FDA Breakthrough Therapy and Fast Track designations by the FDA, and then granted this approval through priority review.
This new treatment, offered by Sanofi Genzyme, was most-recently studied in the COMET phase 3 clinical trial for late-onset patients. That was a non-inferiority trial, meaning the primary goal was to prove that the treatment isn’t inferior to the current standard of care, Myozyme/Lumizyme. That endpoint was met. Over the duration of the trial, the FVC and 6MTW data indicated improvement but wasn’t quite statistically significant. From what we’ve seen in articles written since the 2021 WorldSymposium, there seems to be consensus that this is meaningful improvement.
Nexviazyme is an enzyme replacement therapy designed to more efficiently target the mannose-6-phosphate (M6P) receptor. This is the mechanism by which the enzyme enters the cell and clears excess glycogen. Without clearing that glycogen, it will accumulate to the point of cell damage.
What does this mean for Canadian patients? Approval in the U.S. doesn’t apply to Canada, of course. Each country will make its own decision. Hopefully that process starts soon in all countries so that patients have access to the newest treatment for Pompe disease.