FDA approves Sanofi's Pompe disease treatment

 The U.S. Food and Drug Administration on Friday approved French drugmaker Sanofi's drug, Nexviazyme, for the treatment of patients with Pompe disease, a rare genetic disorder.

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

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

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

https://finance.yahoo.com/news/u-fda-approves-sanofis-pompe-145134099.html