Shire Reports Positive Results From Phase III Studies Of Velaglucerase Alfa

Shire has reported positive results from the first of three phase III studies of velaglucerase alfa, its enzyme replacement therapy for the treatment of Type 1 Gaucher disease.

FDA has accepted the company’s treatment protocol for velaglucerase alfa. In connection to that, Shire has begun its rolling submission of the New Drug Application (NDA) for velaglucerase alfa, allowed under the Fast Track process.

Over 100 patients at 24 sites in ten countries around the world have participated in the clinical studies.

The first trial in the phase III program was a multicenter, randomized, double-blind, two dose study of velaglucerase alfa in patients with Type 1 Gaucher disease. The primary goal was to evaluate the safety and efficacy of velaglucerase alfa in 25 patients with Type 1 Gaucher disease.

Patients were randomized to receive velaglucerase alfa at either 45 U/kg or 60 U/kg for a duration of 12 months.

In the trial, the primary endpoint was reached with patients benefiting from an increase in mean hemoglobin concentration, as compared to baseline after receiving velaglucerase alfa at 60 U/kg IV every other week for 12 months.

A significant improvement compared to baselines were also observed in platelet and spleen sizes, and nominally significant improvements were observed in liver size at this dose. Results were clinically important as defined by standard criteria and consistent with the previously published phase I/II data.

At the 45 U/kg IV dose, significant improvements in hemoglobin, platelet count, and spleen volume were also demonstrated. The magnitude of changes in the 45U/kg dose was also clinically important, and a trend in liver volume reduction was observed. The 60U/kg dose performed numerically as well or better than 45U/kg across all measured clinical endpoints.

Sylvie Gregoire, president of Shire, said: “We are very pleased with the progress of the velaglucerase alfa program from both a clinical and regulatory perspective. This data are consistent with those previously reported from the Phase I/II and extension studies. We will continue to work diligently with the FDA and other regulatory agencies to make velaglucerase alfa available as soon as possible to help meet the needs of the Gaucher community.”