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news.Shire, a global specialty biopharmaceutical company, has reported positive data from a Phase III clinical trial (TKT-034) designed to assess the safety of switching to VPRIV (velaglucerase alfa for injection), from imiglucerase, and an interim analysis of safety data from an continuing multicenter open-label treatment protocol (HGT-GCB-058) implemented to provide VPRIV to patients suffering from continuing imiglucerase shortage.
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Velaglucerase alfa is manufactured in Shire’s facility in Cambridge MA, which was inspected and approved by the FDA for the commercial production of VPRIV.
VPRIV was approved by the US FDA as a long-term enzyme replacement therapy for adult and pediatric patients with Type 1 Gaucher disease on February 26, 2010.
Shire claimed that a post-hoc analysis of Phase I/II data added to the growing body of clinical evidence which support the use of VPRIV in patients both switching from imiglucerase or who are treatment naive.
A post hoc analysis from a third study, TKT-025EXT, designed to evaluate attainment of long-term therapeutic goals in 8 patients with Type 1 Gaucher disease treated with velaglucerase alfa, was also presented by the company.
The initial dose of 60 U/kg was lowered to 30 U/kg after patients achieved at least 2 of 4 predefined therapeutic goals following 1 year of treatment. Clinically meaningful achievement of long-term therapeutic goals for hemoglobin concentration, platelet counts, and liver and spleen volumes was observed within 4 years of initiation of treatment.
Adult and pediatric patients with Type 1 Gaucher disease were transitioned from imiglucerase (15-60 U/kg every other week) to the same number of units of VPRIV in the Phase III switch study (40 patients) and the ongoing US treatment protocol (>150 patients). In study TKT-034, no patients developed IgG antibodies to VPRIV, including 3 patients who tested positive for anti-imiglucerase antibodies at screening.
Additionally, hemoglobin concentration, platelet counts, and liver and spleen volumes remained unchanged over the course of the one year study, establishing safety and maintenance of efficacy over this time frame. One patient in the Phase III trial discontinued due to a serious hypersensitivity reaction and the most common side effects reported in the two studies were infusion-related reactions.
Dr Gregory Grabowski, director of the Division of Human Genetics, Cincinnati Children’s Hospital Medical Center and principal investigator of the 034 study, said: “Results from the Phase III study provide important information regarding the safety and sustained efficacy of VPRIV for patients with Type 1 Gaucher disease who were previously on imiglucerase and should help inform treatment decisions during and after the imiglucerase supply shortage. These data confirm what many physicians have experienced.”
Shire also reported key findings that indicated significant antigenic differences when antibody response to treatment with VPRIV and imiglucerase were compared. Among the 99 patients who enrolled in the Phase III studies, the seroconversion rate was 1% (1 of 82) in contrast to VPRIV versus 23% (4 of 17) against imiglucerase.