The US Food and Drug Administration (FDA) has approved Sanofi Genzyme’s avalglucosidase alfa-ngpt (Nexviazyme®), an enzyme replacement therapy (ERT), for the treatment of late-onset Pompe disease (LOPD) in patients who are at least 1 year old.

Avalglucosidase alfa helps reduce the accumulation of glycogen that occurs in the skeletal and heart muscles of patients with Pompe disease. The accumulation, which stems from an enzyme deficiency, results in muscle weakness and leads to premature death from respiratory or heart failure.

“Pompe disease is a debilitating and progressive condition that significantly inhibits mobility and breathing,” said Bill Sibold, executive vice president of Sanofi Genzyme, describing the therapy as “a potential new standard of care for people living with late-onset Pompe disease.”


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In a study of 100 patients, avalglucosidase alfa and alglucosidase alfa (Lumizyme®), another ERT approved by the FDA for Pompe disease, led to similar improvements in lung function, according to an FDA statement. It has been shown to improve walking distance measures in patients with LOPD, a statement from Sanofi said.

The treatment is given intravenously as a monotherapy every 2 weeks at a dosage of 20 mg/kg for patients ≥30 kg or 40 mg/kg for patients <30 kg. Sanofi officials said it is expected to be available in the US in the coming weeks.

The FDA previously gave its orphan drug designation to avalglucosidase alfa and granted Fast Track, Priority Review, and Breakthrough Therapy designations to the application for it.

Reference

FDA approves new treatment for Pompe disease. News release. US Food and Drug Administration; August 6, 2021.

FDA approves Nexviazyme® (avalglucosidase alfa-ngpt), an important new treatment option for late-onset Pompe disease. News release. Sanofi; August 6, 2021.