FDA approves new treatment for Pompe disease

Today, the United States Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion in patients 1 year of age and older with late-onset Pompe disease.

Patients with Pompe disease have an enzyme deficiency which causes a complex sugar called glycogen to build up in the skeletal and heart muscles, which causes muscle weakness and premature death from respiratory or heart failure. Normally, glycogen – the stored form of glucose – breaks down to release glucose into the bloodstream to be used as fuel for cells.

“Pompe disease is a rare genetic disorder 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 Medicine and reproduction at the FDA’s Center for Drug Evaluation. and Research. “Today’s approval offers 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 new, additional, effective and safe therapies for rare diseases, including Pompe disease. “

Nexviazyme, an enzyme replacement therapy, is an intravenous drug that helps reduce the buildup of glycogen. The efficacy of Nexviazyme for the treatment of Pompe disease was demonstrated in a study involving 100 patients randomized to take Nexviazyme or another FDA approved enzyme replacement therapy for Pompe disease. Treatment with Nexviazyme improved lung function similarly to the improvement seen with the other treatment.

The most common side effects included headache, fatigue, diarrhea, nausea, joint pain (arthralgia), dizziness, muscle pain (myalgia), itching (pruritus), vomiting, difficulty breathing (dyspnea), redness of the skin (erythema), a feeling of “pins and needles” (paraesthesia) and skin welts (hives). Serious reactions included hypersensitivity reactions such as anaphylaxis and infusion-associated reactions including respiratory distress, chills and increased body temperature (pyrexia). Patients who may have fluid overload or whose cardiac or respiratory function is compromised may be at risk for severe acute cardiorespiratory failure.

The FDA has granted this application the designations of Fast Track, Priority Review and Breakthrough Therapy. Nexviazyme has also received an orphan drug designation, which offers incentives to help and encourage drug development for rare diseases. The FDA has granted approval for Nexviazyme to Genzyme Corporation.

###

---