FDA Approves Genzyme’s Nexviazyme for Pompe Disease

The FDA has approved Sanofi subsidiary Genzyme’s Nexviazyme for treatment of patients aged one year and older with late-onset Pompe disease.

The rare inherited disease is caused by an enzyme deficiency that leads to the buildup of glycogen, a complex sugar that the body normally breaks down for energy. The sugar, which builds up in skeletal and heart muscles, can lead to premature death from respiratory or heart failure.

In a study of 100 patients who were randomized to receive Nexviazyme (avalglucosidase alfa-ngpt) or another FDA-approved enzyme replacement therapy, the two treatments showed similar efficacy in improving lung function.