NEW YORK (AP) — Protalix BioTherapeutics Inc. said Thursday a late-stage study of its Gaucher disease drug met its primary goals. The company said patients taking Uplyso had significantly smaller spleen volume, which is a symptom of Gaucher disease, a rare enzyme disorder that can cause liver and neurological problems. Patients also had greater hemoglobin levels and decreased liver size. The study tested two doses of intravenous Uplyso on 31 patients around the world. Protalix said patients who received the larger dose had a significant reduction in blood platelet counts. Patients who were given the smaller dose had lower blood platelet levels, but the improvement was not statistically significant. Patients in the nine-month trial received a dose of Uplyso every two weeks. The doses were either 30 milligrams per kilogram of the patient’s body weight, or 60 milligrams per kilogram. Protalix said both patient groups had significantly lower spleen volumes after six months of treatment, and there were no serious side effects in the trial. Two of the 31 patients experienced hypersensitivity, and two developed antibodies to the drug, it said. The Israeli company plans to complete a filing for Food and Drug Administration approval before the end of 2009. It had been developing Uplyso under the name prGCD.
