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 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.