FDA Approves New Drug for Late-Onset Pompe Disease


Avalglucosidase alfa-ngpt is an enzyme replacement therapy that reduces glycogen accumulation.

The US Food and Drug Administration (FDA) has approved avalglucosidase alfa-ngpt (Nexviazyme) as an intravenous infusion treatment for patients with late-onset Pompe disease at least 1 years old.

The approval was awarded to Genzyme Corporation.

The Disease

Patients with this disorder have an enzyme deficiency that can lead to the accumulation of glycogen in skeletal and heart muscles, which can cause muscle weakness and premature death from respiratory or heart failure.

The intravenous treatment is an enzyme replacement therapy that helps reduce glycogen accumulation.

The Study

The approval is based on the results of a 100-patient randomized controlled trial in which avalglucosidase alfa-ngpt resulted in improved lung function similar to what is seen with other therapies.


Some common side effects included headache, fatigue, diarrhea, nausea, arthralgia, dizziness, myalgia, pruritus, vomiting, dyspnea, erythema, paresthesia, and urticaria.

There were also various serious reactions associated with the medication, including hypersensitivity reactions like anaphylaxis and infusion-associated reactions such as respiratory distress, chills, and pyrexia.

The treatment was previously granted Fast Track, Priority Review and Breakthrough Therapy designations by the FDA, as well as orphan drug designation.


“Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives,” said Janet Maynard, MD, deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research, in a statement. “Today’s approval brings patients with Pompe disease another enzyme replacement therapy option for this rare disease.”

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