“This approval is incredible news for patients and their families who are living with this debilitating disease and have limited options,” Brad Crittenden, executive director at the Canadian Association of Pompe said.
Avalglucosidase alfa-ngpt, manufactured by Sanofi Genzyme, is an enzyme replacement therapy administered as an intravenous infusion. The drug targets the mannose-6-phosphate receptor to promote the transport of the lysosomal enzyme acid alpha-glucosidase, the enzyme that is deficient or absent in Pompe disease, into cell lysosomes.
Therefore, the drug enhances the clearance of glycogen in target tissues. The accumulation of glycogen in tissues, such as the diaphragm, cardiac, and skeletal myocytes, is deleterious.
Read more about Pompe disease experimental therapies
“Due to the severity of Pompe disease, it significantly affects patients’ mobility and breathing, things that we often take for granted,” Mark Tarnopolsky, MD, PhD, FRCPC, director of the Neuromuscular and Neurometabolic Clinic at McMaster University Medical Center in Ontario, Canada said.
“The Health Canada approval of Nexviazyme is an important milestone for Canadian Pompe patients and may represent a new standard of care.”
Avalglucosidase alfa-ngpt was approved on August 6, 2021, by the US Food and Drug Administration (FDA) for the treatment of patients with LOPD aged 1 year and older. The approval followed the publication of positive results demonstrating its efficacy and safety.
“While no cure currently exists, it’s important to be able to provide new treatment options and continued research for Canadian patients living with Pompe disease,” Carrie McElroy, interim general manager for Sanofi Genzyme said.
Nexviazyme™ (avalglucosidase alfa for injection) is now approved in Canada for patients with late-onset Pompe disease (acid α-glucosidase deficiency). News release. Sanofi Canada; November 15, 2021.