FDA Approves Pfizer’s Elelyso To Treat Form of Gaucher Disease

The Food and Drug Administration May 1 approved Elelyso (taliglucerase alfa) for long-term enzyme replacement therapy to treat a form of Gaucher disease, a rare genetic disorder.

Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase, the agency said. The enzyme deficiency causes fatty materials (lipids) to collect in the spleen, liver, kidneys, and other organs. The major signs of Gaucher disease include liver or spleen damage, low red blood cell counts (anemia), low blood platelet counts, and bone problems.

Elelyso is an injection that replaces the missing enzyme in patients with a confirmed ...