Priya Kishnani, MD, Professor of Pediatrics at the Duke University School of Medicine, discusses the latest long-term results from the COMET study involving late-onset Pompe disease.
Pompe disease is a lysosomal disorder caused by inadequate levels of the enzyme acid alpha-glucosidase (GAA) which results in the accumulation of glycogen in the body. Recently, the US Food and Drug Administration approved a second enzyme replacement therapy (ERT), avalglucosidase alfa, for the treatment of persons with Pompe disease. That approval was largely based on the 49-week COMET trial, a phase 3 non-inferiority clinical trial comparing avalglucosidase alfa vs alglucosidase alfa in patients with late-onset Pompe disease.
The COMET study has continued and Dr. Kishnani describes the latest results that have followed the patients for over 145 weeks. This data was recently presented at WORLDSymposium 2023 in Orlando FL.
As discussed by Dr. Kishnani, Following the 49-week primary analysis phase of the study, participants could enter an extended treatment phase. All 51 who received avalglucosidase alfa (20 mg/kg every 2 weeks) continued this in the extended treatment phase. Of 49 who received alglucosidase alfa, 44 entered the extended treatment phase and switched to avalglucosidase alfa. Trends for improvement or stabilization from baseline to week 145 were observed for primary and secondary outcomes of respiratory and motor function. Only five participants discontinued treatment due to 6 treatment-emergent adverse events.
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