Genetic disorders

FDA Approves New Pompe Disease Treatment Option

The FDA has approved Nexviazyme (avalglucosidase alfa-ngpt) for the treatment of patients with late-onset Pompe disease aged 1 year or older.

The enzyme replacement therapy is given as intravenous infusion and helps to reduce glycogen accumulation in these patients. Its safety was examined in a study of 100 patients randomized to receive either Nexviazyme or another FDA-approved enzyme replacement therapy for Pompe disease. Results with Nexviazyme were similar to those seen with the other treatment option.

The most commonly reported side effects included headache, fatigue, diarrhea, nausea, arthralgia, dizziness, myalgia, pruritus, vomiting, dyspnea, erythema, paresthesia and urticaria. Serious reactions included anaphylaxis, respiratory distress, chills, and pyrexia.

Michael Potts


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