With its recent approval of Nexviazyme (avalglucosidase alfa-ngpt), Sanofi (NSDQ:SNY) has asserted its control over the Pompe disease landscape.
In 2006, FDA approved Genzyme’s Myozyme (alglucosidase alfa) for Pompe disease. Sanofi acquired the company a decade ago. Additionally, FDA approved Lumizyme in 2010, which uses the same biologic, but is indicated for patients eight years of age or older. The company uses a larger bioreactor to make Lumizyme.
Nexviazyme is indicated for patients one year of age or older.
Pompe disease results from a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase (GAA), which triggers a surplus of glycogen in muscles.
Nexviazyme targets a pathway used to transport GAA to lysosomes in cells known as the mannose 6-phosphate receptor (M6P).
The drug resulted in a roughly 15-fold increase in M6P levels compared to alglucosidase alfa in the Phase 3 study. The double-blind COMET trial enrolled 100 treatment-naïve patients, with 51 receiving Nexviazyme and the remainder receiving alglucosidase alfa. Investigators administered the intravenous drug at two-week intervals.
Patients receiving Nexviazyme in the trial demonstrated improvements in respiratory function and walking distance in a 6-minute walk test.
Patients generally targeted Nexviazyme well in the trial, with 2% reporting serious adverse events compared to 6% among alglucosidase alfa recipients.