People with rare disorder lack key enzyme
WEDNESDAY, Aug. 20, 2014 (HealthDay News) -- Cerdelga (eliglustat) has been approved by the U.S. Food and Drug Administration to treat type 1 Gaucher disease, a rare inherited disorder caused by the body's insufficient production of a key enzyme.
Lack of the enzyme glucocerebrosidase causes fatty deposits in the spleen, liver and bone marrow. Symptoms include liver and spleen enlargement, anemia, low blood platelets and bone problems, the FDA said in a news release.
Gaucher affects about 6,000 people in the United States. Cerdelga is a gelatin capsule that's designed to slow the body's production of abnormal fatty deposits.
Cerdelga was evaluated in clinical studies involving 199 people with type 1 Gaucher disease. The most common side effects were fatigue, headache, nausea, diarrhea, and pain of the back, arms and legs, and upper abdomen.
Cerdelga is produced by Genzyme, based in Cambridge, Mass.
The FDA (http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm410585.htm ) has more about this approval.