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.

“Pompe disease is a debilitating and progressive condition that significantly inhibits mobility and breathing,” said Bill Sibold, executive vice president of Sanofi Genzyme. “For decades, we’ve made it our responsibility to research how to target the M6P receptor, the key pathway for cellular uptake of enzyme replacement therapy. Nexviazyme is a potential new standard of care for people living with late-onset Pompe disease and delivers on our promise to pursue medicines for patients living with rare diseases.”