On May 1, 2012, the U.S. Food and Drug Administration (FDA) approved new orphan drug Elelyso to treat a form of Gaucher disease. The experimental biotech drug was produced by Protalix Biotherapeutics and Pfizer.

Gaucher disease stems from an enzyme deficiency that prevents the breakdown of certain fats in the body. It can cause organ damage or death and affects about one in 50,000 to 100,000 people. It is particularly prevalent among Ashkenazi Jews.