WASHINGTON — The FDA has formally approved the enzyme replacement therapy velaglucerase alfa (VPRIV) — already okayed for pre-approval marketing — for pediatric and adult patients with Gaucher disease.  Gaucher disease is a rare genetic disorder affecting between 1 in 50,000 and 1 in 100,000 in the general population.

It limits production of the enzyme glucocerebrosidase, the lack of which allows fatty lipids to build up in the liver, spleen, bones, bone marrow, and nervous system.  Drug manufacturer Shire had been given the go-ahead in September 2009 to market the drug before formal approval to ease a supply shortage of imiglucerase (Cerezyme), the only drug then approved for Gaucher’s patients. (See New Gaucher Drug Goes to Market During Shortage)  Approval of VPRIV was based on clinical trials involving 82 patients ages 4 and older with Type 1 Gaucher disease, some of whom switched from imiglucerase.  “The approval of VPRIV will provide a safe and effective alternative treatment for patients with Gaucher disease,” Julie Beitz, MD, director of the FDA’s Office of Drug Evaluation III, said in a statement. “Patients who previously received Cerezyme as an enzyme replacement therapy for their Type 1 Gaucher disease can be safely switched to VPRIV.”  The most common adverse reactions to VPRIV are allergic ones, although headache; dizziness; pain in the abdomen, back and joints; nausea; fatigue/weakness; fever; and prolonged activated partial thromboplastin time have also been noted.

Source: www.medpagetoday.com/ProductAlert/DevicesandVaccines/18719