CAMBRIDGE, Mass., Nov. 4, 2010 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, Nasdaq: SHPGY), the global specialty biopharmaceutical company, today presented positive new data from a Phase III clinical trial (study 039) designed to evaluate the efficacy of VPRIV® (velaglucerase alfa for injection) compared with imiglucerase in patients with type 1 Gaucher disease at the 2010 Annual American Society of Human Genetics (ASHG) in Washington, D.C. The study met its primary endpoint and adds to the growing body of clinical evidence which supports the use of VPRIV in patients who have transitioned from imiglucerase or who are treatment-naive.
In the 039 (head-to-head) study, adult and pediatric patients with type 1 Gaucher disease were included in a 9-month, global, randomized, double-blind, non-inferiority study comparing VPRIV with imiglucerase in treatment-naive patients aged >/= 2 years, with anemia and either thrombocytopenia or organomegaly. Patients were randomized in a 1:1 ratio to receive either VPRIV or imiglucerase at a dose of 60U/kg via continuous infusion over one hour every other week for 39 weeks (total of 20 infusions per patient). 35 patients in 9 countries were randomized and 34 received the study drug (intent-to-treat [ITT] population was 17 in both the VPRIV and imiglucerase groups). The per-protocol (PP) analysis included 15 patients in each group. Baseline clinical characteristics were generally similar between the 2 groups, although hemoglobin concentrations appeared slightly higher in the VPRIV group.
After 9 months of treatment, hemoglobin concentration improved in both groups. The estimated mean treatment difference for hemoglobin concentration from baseline between patients treated with VPRIV and imiglucerase was 0.14 and 0.16 g/dL in the ITT and PP populations, respectively, with a lower bound of the 97.5% one-sided confidence interval of –0.60 g/dL in both populations, greater than the pre-defined non-inferiority margin of –1.0 g/dL. These results indicate that the primary endpoint was met. Both the VPRIV and imiglucerase groups showed substantial improvements in the secondary endpoints, including platelet counts, spleen volume, liver volume, and plasma biomarkers with no statistically significant difference demonstrated between the treatment groups. The majority of adverse events were mild or moderate in severity, including one serious adverse event (SAE) seen with VPRIV which was an allergic skin reaction that resolved without sequalae.
Shire also reported important findings that suggested substantial antigenic differences when antibody response to treatment with VPRIV and imiglucerase were compared. No patient treated with VPRIV developed anti-drug antibodies while 4 patients in the imiglucerase group developed antibodies to imiglucerase. Of these four imiglucerase treated patients, 1 patient had antibodies that inhibited enzyme activity in vitro and enzyme uptake in a cell-based assay. 3 patients had antibodies that did not inhibit enzyme activity or uptake.
Data from the Phase III clinical trial extension were also presented at ASHG. In the six month extension study patients from study 034 with type 1 Gaucher disease previously treated with imiglucerase, successfully transitioned to VPRIV and maintained hematological parameters at therapeutic levels through 18 months of continuous treatment. VPRIV was generally well tolerated, and appeared to show a favourable immunogenicity profile.
"For physicians, drug safety is a major consideration; the reported lack of antibodies to infused velaglucerase alfa in these studies, both in naive and switch patients, is re-assuring to patients and their physicians alike," said Gregory M. Pastores MD, Associate Professor of Neurology and Pediatrics at the NYU School of Medicine in New York.
Shire's VPRIV clinical trial program is the largest and most comprehensive set of Phase III trials conducted to date for type 1 Gaucher disease.
More about VPRIV
VPRIV (velaglucerase alfa for injection) was approved by the US FDA as a hydrolytic lysosomal glucocerebroside-specific indicated for long-term enzyme replacement therapy for adult and pediatric patients with type 1 Gaucher disease on February 26, 2010. The European Commission also has granted marketing authorization for VPRIV (velaglucerase alfa), for the long-term treatment of type 1 Gaucher disease on August 26, 2010, making it available in 30 countries across Europe.
VPRIV is for patients who are treatment-naive as well as patients who have previously been treated with imiglucerase.
Important Safety Information
The most serious adverse reactions seen with VPRIV were hypersensitivity reactions. Infusion-related reactions were the most commonly observed adverse reactions in patients treated with VPRIV in clinical studies. The most commonly observed symptoms of infusion-related reactions were: headache, dizziness, low or high blood pressure, nausea, tiredness and weakness, and fever. Generally the infusion-related reactions were mild and, in treatment-naive patients, onset occurred mostly during the first 6 months of treatment and tended to occur less frequently with time.
All adult side effects of VPRIV are considered relevant to children (ages 4 to 17 years). Side effects more commonly seen in children compared with adult patients included: upper respiratory tract infection, rash, aPTT prolonged, and fever. The safety of VPRIV has not been established in patients younger than 4 years of age.
As with all therapeutic proteins, there is a potential for immunogenicity. In the clinical studies 1 of 54 treatment-naive patients treated with VPRIV developed IgG class antibodies. It is unknown if the presence of IgG antibodies to VPRIV is associated with a higher risk of infusion reactions.
VPRIV is not available in all countries and prescribing information may differ between countries. Please consult your local prescribing information. Full prescribing information for VPRIV in the US can be found at www.VPRIV.com.
Notes to editors
Shire's strategic goal is to become the leading specialty biopharmaceutical company that focuses on meeting the needs of the specialist physician. Shire focuses its business on attention deficit hyperactivity disorder (ADHD), human genetic therapies (HGT) and gastrointestinal (GI) diseases as well as opportunities in other therapeutic areas to the extent they arise through acquisitions. Shire's in-licensing, merger and acquisition efforts are focused on products in specialist markets with strong intellectual property protection and global rights. Shire believes that a carefully selected and balanced portfolio of products with strategically aligned and relatively small-scale sales forces will deliver strong results.
For further information on Shire, please visit the Company's website: www.shire.com.
"SAFE HARBOR" STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM ACT OF 1995
Statements included herein that are not historical facts are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, the Company's results could be materially adversely affected. The risks and uncertainties include, but are not limited to, risks associated with: the inherent uncertainty of research, development, approval, reimbursement, manufacturing and commercialization of the Company's Specialty Pharmaceutical and Human Genetic Therapies products, as well as the ability to secure and integrate new products for commercialization and/or development; government regulation of the Company's products; the Company's ability to manufacture its products in sufficient quantities to meet demand; the impact of competitive therapies on the Company's products; the Company's ability to register, maintain and enforce patents and other intellectual property rights relating to its products; the Company's ability to obtain and maintain government and other third-party reimbursement for its products; and other risks and uncertainties detailed from time to time in the Company's filings with the Securities and Exchange Commission.
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