According to a press release from the biopharmaceutical company Sanofi, the company’s investigational treatment avalglucosidase alfa will have its Biologics License Application (BLA) evaluated by the US Food and Drug Administration (FDA) under priority review. The therapy is being developed as a treatment for Pompe disease, a rare disease. The agency aims to make a decision on the BLA by May 18, 2021.
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. 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
Avalglucosidase alfa is designed as a long term enzyme replacement therapy that is intended to enhance the delivery of the enzyme acid alpha-glucosidase to the muscle cells. It targets a receptor called mannose-6-phosphate that plays a critical role in delivering the enzyme. If approved, this new therapy could become a new standard of treatment for Pompe disease. The BLA was accepted by the FDA based on encouraging findings from a phase 2 and a phase 3 clinical trial.
The drug also has a Marketing Authorization Application under review by the EU’s European Medicines Agency. In addition, avalglucosidase alfa has earned Promising Innovative Medicine designation from the Medicines and Healthcare Products Regulatory Agency in the UK. With the ongoing progress and review of the therapy, patients in Europe and the US could soon have a new, more effective treatment for their disease next year.
