Protalix BioTherapeutics reported positive Phase III results with Uplyso in treatment-naive patients diagnosed with Gaucher’s disease. The firm expects to submit an NDA before the end of the year.
Uplyso, previously referred to as prGCD, is already being supplied to certain patients under specific guidelines according to the FDA’s expanded-access program. Shire also has a late-stage Gaucher’s therapy that is being marketed under the same program. Both received sanction due to the shortfall in the supply of Cerezyme, Genzyme’s approved medication, after the company’s production was affected when it closed one of its plants due to viral contamination.
Protalix’ trial met its primary endpoint, mean reduction in spleen volume after nine months compared with baselines in both 60 units/kg dose and in the lower 30 units/kg dose treatment groups, Protalix reports. Additionally, the primary endpoint was observed after six months of treatment in both treatment groups.
Statistically significant improvements compared with baselines were also observed in the secondary endpoints including increase in hemoglobin level, decrease in liver size, and increase in platelet count at the 60 units/kg dose, Protalix continues.
Similarly, the 30 units/kg dose also showed statistically significant improvements compared with baselines in hemoglobin level and liver size. Significant nominal elevation in platelet count in this lower dose was also seen, according to the company.
The safety analysis for both doses showed that Uplyso was well tolerated and no serious adverse events were reported. About 6% of patients in the trial developed antibodies to the drug during the study. None of the patients in the trial developed neutralizing antibodies, though. Only 6% of the patients in the trial experienced hypersensitivity.
The Phase III study with Uplyso was a nine-month, randomized, double-blind, parallel-group, dose-ranging evaluation of patients with Gaucher’s disease. Patients were randomized to receive either 60 units/kg or 30 units/kg of the drug administered intravenously once every two weeks. A total of 31 patients were enrolled at 11 centers in Europe, North America, South America, Israel, and South Africa.