U.S. approves drug for patients with Pompe disease


Aug 6 - The U.S. Food and Drug Administration approved the drug Nexviazyme of Sanofi for the treatment of patients with Pompe disease, a rare genetic disorder.

The health regulator allowed enzyme replacement therapy to be used in patients one year and older and with late onset of disease.

Pompe disease, which affects 1 in 40,000 people in the United States is characterized by the build up of a rare sugar called glycogen in skeletal and heart muscles, which can lead to premature death.

Sanofi Genzyme, developed by Nexviazyme, intends to reduce the accumulation of glycogen.