New Enzyme Replacement Therapy Approved for Pompe Disease

New Enzyme Replacement Therapy Approved for Pompe Disease

August 6, 2021 – The U.S. FDA has approved Sanofi’s Nexviazyme^® (avalglucosidase alfa-ngpt), to treat late-onset Pompe disease (LOPD) in patients who are at least a year old.

Pompe is a rare genetic disease that reduces or eliminates the body’s ability to produce acid alpha- glucosidase (GAA). An enzyme needed for proper function of the organs and tissues, GAA is responsible for breaking down a complex sugar called glycogen into a simpler sugar, glucose, that can fuel the body’s cells. In the absence of enough GAA, glycogen accumulates in and damages skeletal and heart muscles.

This can lead to muscle weakness and premature death due to respiratory or heart failure. In a clinical trial, Nexviazyme delivered improvements in respiratory function and walking distance measures for patients diagnosed with LOPD compared to treatment with alglucosidase alfa, a current standard of care. Nexviazyme in an enzyme therapy that can help reduce buildup of glycogen.

Recommended dosing is based on individual patient factors including weight and whether the patient has an adverse reaction. Nexviazyme is administered once every two weeks by intravenous infusion in a facility with appropriate medical support measures are readily available, including cardiopulmonary resuscitation equipment. A black box warning cautions there is a risk of severe hypersensitivity reactions, infusion-associated reactions, and acute cardiorespiratory failure.

Launch and pricing information are not yet available.