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On August 6, 2021, the US FDA announced the approval of Sanofi's enzyme replacement therapy (ERT) Nexviazyme (avalglucosidaseα-ngpt) for the treatment of late-onset Pompe disease (late-onset Pompe disease, LOPD) patients
Pompe disease is caused by a genetic defect or dysfunction of acid alpha-glucosidase (GAA) in the lysosome
The key way to transport the GAA enzyme to the intracellular lysosome is the M6P receptor
▲Defects in glycogen degradation in Pompe disease patients (picture source: pompe.
This approval is based on the positive results obtained from the pivotal phase 3 clinical trial COMET.
At week 49, the trial reached its primary endpoint of improving respiratory function
Note: The original text has been deleted
Reference materials:
[1] FDA approves Nexviazyme® (avalglucosidase alfa-ngpt), an important new treatment option for late-onset Pompe disease.
[2] FDA Approves New Treatment for Pompe Disease.
