(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."
SOURCE Shire Pharmaceutical