Priya Kishnani, MD, Professor of Pediatrics at the Duke University School of Medicine, discusses the latest results from the COMET study assessing the efficacy of a new enzyme replacement therapy (ERT) to treat late-onset Pompe disease.

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, one ERT is approved to treat this condition, alglucosidase alfa. The COMET trial is a phase 3 non-inferiority clinical trial comparing avalglucosidase alfa vs alglucosidase alfa in patients with late-onset Pompe disease. 

In the 49 week study, patients 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 (p=0.0074) was met. 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. 

The drug is currently under review by the U.S. Food and Drug Administration (FDA) with a decision expected on or before May 18, 2021. 

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