FDA Approves New Treatment for Pompe Disease

On August 6th, 2021, FDA announced that they had approved Nexviazyme for intravenous infusion to treat patients one year or older with Pompe Disease. It has been granted Fast Track, Priority Review, and Breakthrough Therapy designations, in addition to an orphan drug designation.

Pompe Disease is a condition that causes excess build-up of glycogen (which is typically broken down into glucose and released into the bloodstream to provide energy to cells) in skeletal and heart muscles, according to the agency. This causes muscle weakness and can lead to premature death from respiratory or heart failure.

Nexviazyme is an intravenous drug designed to reduce glycogen levels. A study was done wherein 100 patients with Pompe’s Disease were randomly given either Nexviazyme or another FDA approved enzyme-replacement therapy. The results found similar levels of efficacy between Nexviazyme and the approved therapy.

“Today’s approval brings patients with Pompe disease another enzyme replacement therapy option for this rare disease,” said Janet Maynard, deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in a press release. “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.”

Source: FDA