The Food and Drug Administration (FDA) has approved avalglucosidase alfa (Nexviazyme) for the treatment of late-onset Pompe disease in patients 1 year of age and older.
Pompe disease is a lysosomal storage disorder caused by inadequate levels of the enzyme acid alpha-glucosidase (GAA) which results in the accumulation of glycogen in the body. Currently, only one ERT is approved to treat this condition, alglucosidase alfa.
The approval was largely based on positive results of the phase 3 COMET trial testing avalglucosidase alfa in late-onset Pompe disease.
As Dr. Kushlaf explains in the above video, the COMET trial was a 49 week study to evaluate the effect of avalglucosidase alfa on respiratory muscle strength as compared to alglucosidase alfa. Participants were randomized to receive avalglucosidase alfa (n=51) or alglucosidase alfa (n=49) infusions every two weeks. The primary study objective, achieving statistical non-inferiority in percent predicted forced vital capacity (FVC%) between the two groups was met (P = .0074). Furthermore, the study showed that treatment with avalglucosidase alfa resulted in greater improvements in upright FVC% predicted at all timepoints and a 2.43% greater increase in FVC% predicted compared to alglucosidase alfa at Week 49. Additionally, the safety profile of avalglucosidase is promising as fewer participants receiving the investigational drug experienced adverse events compared to those in the alglucosidase treatment arm.
To stay up to date with the latest FDA approvals, sign up for our newsletter here.