Possible Treatment for Pompe Disease to be Evaluated Under Priority Review

According to a press release from the biopharmaceutical company Sanofi, the Biologics License Application (BLA) for its drug avalglucosidase alfa has been accepted by the US Food and Drug Administration (FDA) for priority review. This drug is being developed as a treatment for Pompe disease, a rare disease. Under priority review, the agency will make a decision on the application 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 classified as a long term enzyme replacement therapy that aims to boost the delivery of the acid alpha-glucosidase enzyme to the muscles of the patient. This therapy targets a receptor called mannose-6-phosphate (M6P) in order to approve the clearance of glycogen and improve uptake of the enzyme in the muscle cells. Approval of this drug could improve the effectiveness of treatment and set a new standard of care for Pompe disease. The BLA is based on findings from a phase 2 and a phase 3 clinical trial.

Avalglucosidase alfa also has a Marketing Authorization Application under review in the EU. It has also earned Promising Innovative Medicine designation from the UK’s Medicines and Healthcare Products Regulatory Agency. Patients in Europe and the US may soon have access to a new breakthrough treatment for their disease next year.

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