FDA approves Nexviazyme for late-onset Pompe disease

The FDA today approved Nexviazyme, an IV infusion, for the treatment of late-onset Pompe disease among patients aged 1 year and older, according to a press release.

Nexviazyme (avalglucosidase alfa-ngpt, Sanofi Genzyme) is an IV, enzyme replacement therapy that helps decrease glycogen buildup, which accumulates in skeletal and heart muscles among people with Pompe disease and results in muscle weakness and premature death due to respiratory or heart failure. Researchers evaluated the efficacy of Nexviazyme in a study of 100 patients who were randomly assigned to either Nexviazyme or another FDA-approved enzyme replacement for the disease. Study results demonstrated that Nexviazyme improved lung function in a manner like that seen with the other therapy, according to the press release from the FDA.

Common adverse events included headache, fatigue, diarrhea, nausea, arthralgia, dizziness, myalgia, pruritus, vomiting, dyspnea, erythema, paresthesia and urticaria. Serious reactions included hypersensitivity reactions, such as anaphylaxis and infusion-associated reactions, including respiratory distress, chills and pyrexia. The press release noted that patients who are susceptible to fluid volume overload or have compromised cardiac or respiratory function may be at risk for serious acute cardiorespiratory failure.

Nexviazyme received fast track, priority review and breakthrough therapy designations from the FDA, as well as an orphan drug designation.

“Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives,” 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, said in the press release. “Today’s approval brings patients with Pompe disease another enzyme replacement therapy option for this rare disease. The FDA will continue to work with stakeholders to advance the development of additional new, effective and safe therapies for rare diseases, including Pompe disease.”