21 Mar 2011
Shire Presents Data at ACMG for Fabry and Gaucher Patients Who Switched to Replagal® (agalsidase alfa) and VPRIV® (velaglucerase alfa for injection)
New data reported from Fabry and Gaucher clinical studies
Cambridge, MA, US – March 21, 2011 – Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, presented new data for Replagal® (agalsidase alfa) in patients with Fabry disease, and VPRIV® (velaglucerase alfa for injection) in patients with type 1 Gaucher disease, at the 2011 American College of Medical Genetics’ (ACMG) Annual Clinical Genetics Meeting, held in Vancouver, BC, from March 16-20, 2011.
Data from clinical programs evaluating the safety of switching from Fabrazyme® (agalsidase beta) to Replagal and long-term safety and efficacy of switching from Cerezyme® (imiglucerase) to VPRIV were presented.
Preliminary data from Shire's Replagal study 059, an ongoing, multicenter, open-label clinical study, was presented for the first time. This data, following 3 months of treatment, suggests that switching from Fabrazyme to Replagal 0.2 mg/kg every other week is generally well tolerated. This ongoing study includes 70 Fabry patients in the US under Shire's treatment protocol who were switched to Replagal during the continuing Fabrazyme supply shortage. The safety events observed in this study were similar to those seen historically in patients treated with Replagal.
Data from the 52 patients in Shire’s VPRIV study 044, the extension study for studies 034 and 039, were presented. Data from study 058, Shire’s treatment protocol in the US, in 205 type 1 Gaucher patients who switched from Cerezyme were also presented. Taken together, these studies include data from ongoing treatment in more than 250 patients, representing the largest and most comprehensive set of studies to date for Gaucher disease. This collection of long-term data (up to 2 years) adds to the growing body of clinical evidence which supports the use of VPRIV in patients who have previously been treated with imiglucerase.
”The data presented here reinforces our confidence in the treatments that Shire has developed for Fabry and Gaucher diseases. I am pleased that Shire has been able to continue supporting the Fabry and Gaucher patient communities,” commented Dr. Paul M. Fernhoff, Division of Medical Genetics of the Emory University School of Medicine.
Replagal and VPRIV were developed by Shire using its proprietary gene activation technology in human cell lines. Shire has utilized this technology to manufacture enzyme replacement therapies for nearly two decades.
“We are pleased to continue our commitment to support patients and healthcare providers who are dedicated to treating rare diseases. The data presented at ACMG strengthens the body of evidence for Replagal and VPRIV as treatments that may benefit Fabry and Gaucher patients,” said Sylvie Grégoire, President of Shire HGT. “Shire has invested more than $200 million on enhancing capacity and technology in order to ensure safe and consistent drug supplies, and we anticipate the first approval of the new Lexington manufacturing facility at the end of 2011.”
About REPLAGAL (agalsidase alfa)
Replagal is a human form of enzyme alpha-galactosidase A (a-Gal A) manufactured in a human cell line by gene activation. 2011 marks our 15th year of clinical experience with Replagal, which is now approved in 46 countries. Over 2,300 patients are now being treated with Replagal globally. This figure represents about 70 percent of the treated Fabry patients worldwide. Replagal is not currently approved for commercial sale in the U.S.
Replagal is the only human-cell-line-derived form of enzyme replacement therapy (ERT) that is indicated for the long-term treatment of patients with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency).
About VPRIV (velaglucerase alfa for injection)
VPRIV was approved by the US FDA as a hydrolytic lysosomal glucocerebroside-specific indicated for long-term ERT for adult and pediatric patients with type 1 Gaucher disease on February 26, 2010. The European Commission also has granted marketing authorization for VPRIV for the long-term treatment of type 1 Gaucher disease on August 26, 2010, making it available in 30 countries across Europe. Over 1000 patients are now being treated with VPRIV globally. This figure represents about 18 percent of the treated Gaucher patients worldwide.
VPRIV is for patients who are treatment-naïve as well as patients who have previously been treated with imiglucerase.
Replagal Important Safety Information
The most serious adverse reactions seen with Replagal were hypersensitivity reactions. Infusion-related reactions were the most commonly observed adverse reactions in patients treated with Replagal in clinical studies. Most side effects are mild to moderate and include headache, tingling, numbness, tremors, fatigue, change in temperature sensation, increased blood pressure, upset stomach, diarrhea, coughing, sore throat, difficulty sleeping, change in the taste of food, change in smell, difficulty speaking, acne, dry skin and eye problems. About 1 out of 10 patients may have a reaction during or shortly after infusion of Replagal. These effects include chills and facial flushing (warmth and redness).
As with all therapeutic proteins, there is a potential for immunogenicity. IgG antibodies appeared to develop following approximately 3 to 12 months of treatment. After 12 to 54 months of therapy, 17% of Replagal treated patients were antibody positive whereas 7% showed evidence for the development of immunologic tolerance, based on the disappearance of IgG antibodies over time. No IgE antibodies have been detected in any patient receiving Replagal.
Replagal is not available in all countries and prescribing information may differ between countries. Please consult your local prescribing information.
VPRIV 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-naïve 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-naïve 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.
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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.
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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.