FDA approves Nexviazyme for late-onset Pompe disease

The US Food and Drug Administration on Friday approved Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion to treat patients one year of age and older with late-onset Pompe disease, from French pharma major Sanofi (Euronext: SAN).

Patients with Pompe disease have an enzyme deficiency that leads to the accumulation of a complex sugar, called glycogen, in skeletal and heart muscles, which cause muscle weakness and premature death from respiratory or heart failure. Normally, glycogen - the stored form of glucose - breaks down to release glucose into the bloodstream to be used as fuel for the cells.