Amicus reports ‘positive’ long-term data from Phase 3 study of AT-GAA

Amicus Therapeutics announced "positive" results from the global Phase 3 open-label extension study to investigate the long-term efficacy and safety of AT-GAA in adult patients with late-onset Pompe disease, an inherited lysosomal disorder caused by an enzyme deficiency that leads to the accumulation of glycogen in cells. Study participants treated with AT-GAA for up to 104 weeks showed persistent and durable effects on six-minute walk distance, stability in forced vital capacity, and continued reductions in biomarkers of muscle damage and disease substrate. These clinical results are being featured at the 19th Annual WORLDSymposium 2023 in a poster presentation and an oral platform presentation scheduled for Sunday, February 26, 2023, at 9:00 a.m. ET. The presentation will be given by Benedikt Schoser, MD, Department of Neurology, Ludwig-Maximilians-University of Munich and Principal Investigator in the PROPEL study. Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc., stated, "These open label extension data from our Phase 3 PROPEL study of AT-GAA continue to represent meaningful and durable improvements in functional outcomes, as well as persistent reductions in key biomarkers of muscle damage and disease substrate out to two years. These results give great hope that AT-GAA has the potential to become the new global standard of care for people living with Pompe disease."