FDA Approves Nexviazyme For Treatment Of Pompe Disease

(RTTNews) - The U.S. Food and Drug Administration Friday said it has approved Genzyme Corporation's Nexviazyme for treatment of patients 1 year of age and older with late-onset Pompe disease.

Genzyme, is the specialty care global business unit of Sanofi, focused on rare diseases, rare blood disorders, neurology, immunology, and oncology.

"Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people's lives," said Janet Maynard, M.D., deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA's Center for Drug Evaluation and Research.

Patients with Pompe disease have an enzyme deficiency that leads to the accumulation of a complex sugar, called glycogen, in skeletal and heart muscles, which cause muscle weakness and premature death from respiratory or heart failure.

Nexviazyme, an enzyme replacement therapy, is an intravenous medication that helps reduce glycogen accumulation. Treatment with Nexviazyme improved lung function similar to the improvement seen with the other therapy.

The most common side effects included headache, fatigue, diarrhea, nausea, joint pain, dizziness, muscle pain, itching, vomiting, difficulty breathing, skin redness, feeling of "pins and needles" and skin welts.

Nexviazyme also received an orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.