Data for an Experimental Pompe Disease Therapy to be Presented at Upcoming Conference

According to a press release from Sanofi, platform and poster presentations focused on the company’s investigational product candidate avalglucosidase alfa are slated to be made public at the 17th annual WORLDSymposium™, which is scheduled for February 8-12, 2021. This experimental treatment is being tested as a treatment for the infantile and late-onset forms of Pompe disease, a rare disease. Sanofi will also present on some of its other experimental therapies as well at this event.

About Pompe Disease

Pompe disease, which is also known as glycogen storage disease type II, is a genetic, metabolic disorder. This disease can cause damage to the nerves and muscles throughout the body, and is the result of the excessive buildup of glycogen in the cellular lysosome. This occurs because of the deficiency of a certain enzyme. The disease is the result of a genetic mutation that appears on chromosome 17. Symptoms of Pompe disease vary depending on when it appears. They can include poor growth, trouble feeding, enlarged heart, poor muscle tone, muscle weakness, and breathing problems. There is also a late onset form that mostly differs by the absence of heart abnormalities. The primary treatment for Pompe disease is enzyme replacement therapy. While this treatment can improve symptoms and survival, a high dosage is necessary, and it primarily only halts disease progression. To learn more about Pompe disease, click here.

About Avalglucosidase Alfa

A total of seven presentations related to the company’s research in Pompe disease are scheduled. Avalglucosidase alfa is in development as an enzyme replacement therapy, designed to deliver the enzyme that is deficient in patients living with the disease: acid α-glucosidase (GAA). The mechanism of action delivers GAA directly into the lysosomes of the muscle cells. This occurs through the targeting of a receptor called mannose-6-phosphate (M6P), which is essential for transporting GAA. In comparison to currently used enzyme replacement therapies, avalglucosidase alfa is designed with 15 times the M6P content.

The US Food and Drug Administration (FDA) previously accepted the Biologics License Application (BLA) for this therapy in November of last year, under Priority Review protocols. The agency is anticipated to make a decision on the application by May 2021. Avalglucosidase alfa has also earned Fast Track designation and Breakthrough Therapy designation from the FDA.

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