Positive results from Shire's velaglucerase alfa Phase III study for Type 1 Gaucher disease

Feb 12 2010

Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, today 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."