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News Release - June 14, 2007
Shire's ELAPRASE(TM) (idursulfase) Approved by Health Canada for Treatment of Hunter SyndromeBASINGSTOKE, England and CAMBRIDGE, Massachusetts, June 14 (HSMN NewsFeed) -- Shire plc (LSE: SHP, NASDAQ: SHPGY, TSX: SHQ) announces that Health Canada (under priority review) has approved ELAPRASE, a human enzyme replacement therapy for the treatment of Hunter syndrome, for sale and marketing in Canada. Hunter syndrome, also known as Mucopolysaccharidosis II (MPS II), is a rare, life-threatening genetic condition mainly affecting males that results from the absence or insufficient levels of the lysosomal enzyme iduronate-2-sulfatase. Without this enzyme, cellular waste products accumulate in tissues and organs, which then begin to malfunction.
ELAPRASE has been made available on a limited basis to Canadian patients since January 2007 through Health Canada's Special Access Program (SAP) but will now be available on a more widespread basis across the nation. Health Canada's approval follows the July 2006 marketing approval of ELAPRASE by the U.S. Food and Drug Administration and the January 2007 marketing authorization of ELAPRASE by the European Commission. At the end of the first quarter 2007, 291 patients are being treated with ELAPRASE worldwide. Shire estimates that there are approximately 2,000 patients worldwide afflicted with Hunter syndrome in areas where reimbursement may be possible.
"Health Canada's approval of ELAPRASE is another important step in bringing this much-needed treatment to Hunter syndrome patients around the world," said Matthew Emmens, chief executive officer of Shire. "Also at this time we want to thank the Canadian patients for their participation in the ELAPRASE clinical trials; without their commitment and determination, we would not have been able to bring this treatment to Canada and others would continue to suffer the debilitating symptoms of Hunter syndrome."
According to Dr. Lorne Clarke, Medical Director of the Provincial Medical Genetics Program and researcher at the University of British Columbia, "The approval of ELAPRASE is an exciting advancement. There is potential to make a significant improvement in this progressive disorder by treating patients early."
Clinical Trial Results
A 53-week, randomized, double-blind, placebo-controlled Phase II/III trial demonstrated that ELAPRASE provides clinically important benefits to Hunter syndrome patients. The primary efficacy endpoint of the trial was a composite analysis of changes from baseline in two clinical measures: a 6-minute walk test and percent predicted forced vital capacity. Shire is pleased to report that this endpoint achieved statistical significance compared to placebo. Also, after one year of treatment, patients receiving weekly infusions of ELAPRASE experienced a significant mean increase in the distance walked in six minutes of 35 meters compared to patients receiving placebo. The change in percent predicted forced vital capacity was not statistically significant compared to placebo.
Anaphylactoid reactions, which have the potential to be life threatening, have been observed in some patients treated with ELAPRASE. Patients with compromised respiratory function or acute respiratory disease may be at risk of serious exacerbation of their respiratory dysfunction due to infusion related reactions. These patients require additional monitoring. Late-emergent anaphylactoid reactions have been observed after ELAPRASE administration. Patients who have experienced severe and refractory anaphylactoid reactions may require prolonged observation times. Due to the potential for severe infusion reactions appropriate medical support measures should be readily available when ELAPRASE is administered.
In all phases of clinical study for ELAPRASE, 11 patients experienced anaphylactoid reactions during 19 of 8,274 infusions (0.2%) and no patients discontinued treatment permanently as a result of an infusion reaction. The most common adverse events observed in >30% of patients during the Phase II/III trial were pyrexia, headache and arthralgia.
Fifty percent of patients across all studies (53 of 106) developed anti-idursulfase IgG antibodies.
Adverse reactions that were reported during the 53-week placebo-controlled study were almost all mild to moderate in severity.
Studies have not been performed in patients under 5 or over age 65.
ELAPRASE is a purified form of the lysosomal enzyme iduronate-2-sulfatase and is produced by recombinant DNA technology in a human cell line.
Shire Human Genetic Therapies is actively tracking health data among individuals affected by Hunter syndrome as part of the company's long-term outcome survey, called the Hunter Outcome Survey (HOS). HOS is designed to support the gathering, analysis, reporting and sharing of data from around the world about Hunter syndrome. Shire believes that the inclusion of all people affected by Hunter syndrome, whether on therapy or not, and the analysis and dissemination of the information gathered, will allow for better understanding of Hunter syndrome and disease education on a global scale.
More information about ELAPRASE and Hunter syndrome is available through Shire Human Genetic Therapies in Canada at 1-416-322-2886.
About Hunter Syndrome
Hunter syndrome (MPS II) is a serious genetic disorder mainly affecting males that interferes with the body's ability to break down and recycle waste substances called mucopolysaccharides, also known as glycosaminoglycans or GAG. Hunter syndrome is one of several related lysosomal storage diseases.
In Hunter syndrome, cumulative build-up of GAG in cells throughout the body interferes with the way certain tissues and organs function, leading to severe clinical complications and early mortality. Physical manifestations for some people with Hunter syndrome may include distinct facial features, a large head and an enlarged abdomen. People with Hunter syndrome may also experience hearing loss, thickening of the heart valves leading to a decline in cardiac function, obstructive airway disease, sleep apnea, and enlargement of the liver and spleen. In some cases, central nervous system involvement leads to progressive neurologic decline.
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 and hyperactivity disorder (ADHD), human genetic therapies (HGT), gastrointestinal (GI) and renal diseases. The structure is sufficiently flexible to allow Shire to target new therapeutic areas to the extent opportunities arise through acquisitions. Shire believes that a carefully selected portfolio of products with a strategically aligned and relatively small-scale sales force will deliver strong results.
Shire's focused strategy is to develop and market products for specialty physicians. Shire's in-licensing, merger and acquisition efforts are focused on products in niche markets with strong intellectual property protection either in the US or Europe.
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, Shire's results could be materially affected. The risks and uncertainties include, but are not limited to, risks associated with: the inherent uncertainty of pharmaceutical research, product development, manufacturing and commercialization; the impact of competitive products, including, but not limited to the impact of those on Shire's Attention Deficit and Hyperactivity Disorder (ADHD) franchise; patents, including but not limited to, legal challenges relating to Shire's ADHD franchise; government regulation and approval, including but not limited to the expected product approval dates of SPD503 (guanfacine extended release) (ADHD) and SPD465 (extended release triple-bead mixed amphetamine salts) (ADHD); Shire's ability to secure new products for commercialization and/or development; Shire's ability to benefit from its acquisition of New River Pharmaceuticals Inc.; and other risks and uncertainties detailed from time to time in Shire plc's filings with the Securities and Exchange Commission, particularly Shire plc's Annual Report on Form 10-K for the year ended December 31, 2006.
Source: Shire PLC
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