Shire plc presented positive results from its first Phase III study (TKT 032) evaluating safety and efficacy of velaglucerase alfa, its investigational enzyme replacement therapy for the treatment of Type 1 Gaucher disease. The data were presented in an oral presentation at the Lysosomal Disease Network (LDN) World Symposium in Miami, Florida. Data from a pediatric subgroup of this study and five year follow-up results from a long-term Phase I/II extension study (TKT025 EXT) conducted in adults were also reported and add to the available data on the long-term safety and efficacy of velaglucerase alfa in patients with Type 1 Gaucher disease.
Both studies (TKT032 and TKT025 EXT) met their primary end-points.
Additionally, Shire reported important findings from a study that compared patient antibody response to velaglucerase alfa and imiglucerase. All patients enrolled in the velaglucerase alfa Phase III clinical studies underwent a comprehensive panel of tests that were developed and validated to assess antibody response. In each study, samples were first screened using an electrochemiluminescence (ECL) assay. Positive samples were confirmed using a quantitative radioimmunoprecipitation (RIP) assay. Positive cut-points were established for the ECL assay as 5 ng/mL as well as in terms of fixed raw counts, and for the RIP assay as 4 ng/mL. The results suggest significant antigenic differences between velaglucerase alfa and imiglucerase, with only 1% seroconversion rates against velaglucerase alfa.
“The combined data presented today provides additional and compelling support for the long-term clinical efficacy and safety of velaglucerase alfa,” said Pramod Mistry, MD, PhD, FRCP, Professor of Pediatrics & Internal Medicine at Yale University School of Medicine. “The Gaucher community is very fortunate to have velaglucerase alfa as an option for patients.”
Date: February 11, 2010
Source: Shire plc
Filed Under: Drug Discovery
