Healthcare Industry News: Mucopolysaccharidosis
News Release - January 11, 2007
The European Commission Approves First and Only Enzyme Replacement Therapy for Hunter SyndromeBASINGSTOKE, England, January 11 (HSMN NewsFeed) -- The European Commission has granted marketing authorisation for the use of idursulfase (ElapraseŽ), for the long-term treatment of patients with Hunter syndrome. Idursulfase, marketed by Shire Human Genetics Therapies, is the first and only enzyme replacement treatment for people suffering from Hunter syndrome (Mucopolysaccharidosis II) since this condition was identified 100 years ago.
Idursulfase, which is delivered by weekly infusions, replaces the deficient or missing enzyme, iduronate-2-sulfatase (I2S), that people with this progressive and debilitating genetic disorder fail to produce in sufficient quantities. This lack of enzyme interferes with the body's ability to break down and recycle complex carbohydrates, common within secretions and cellular connective tissues, called glycosaminoglycans (GAG). The build up of GAG causes damage to the body's tissues, which can result in hearing loss, declining heart function, an enlarged liver and spleen and a limited range of motion and mobility as a consequence of joint stiffness[2-5]. Treatment with idursulfase leads to improved endurance as measured by the 6 minute walk test. The clinical trial also showed significant improvement in spleen and liver size and urine GAG levels[1, 7, 8].
Hunter syndrome is a very rare, progressive and life-threatening condition, which primarily affects males. It is one of several hereditary metabolic diseases, known collectively as lysosomal storage disorders, the symptoms of which start to become noticeable after the first two years of life. The life expectancy for the more severely affected is only 10-20 years.
"Patients with Hunter syndrome have a very poor long-term prognosis. The approval of idursulfase means that for the first time in the history of managing this disorder, we have a treatment to offer our patients, which targets the underlying problem of this disease and improve symptoms," said Professor Michael Beck from the University Clinic of Mainz, Germany, specialist in Mucopolysaccharidosis (MPS) disorders. "The safety and efficacy data from clinical studies are promising and demonstrate the potential of this therapy to completely change the approach to Hunter syndrome treatment to the overall management and control of many of the symptoms of the disease."
"This therapy offers the first real hope of long-term treatment for patients with Hunter syndrome, offering them the chance for an improvement in their symptoms and so their day-to-day life," said Christine Lavery from MPS Society. "This is a devastating disorder which affects not only the individual, but their families. We hope that all patients with Hunter syndrome in Europe, who will benefit from this treatment, will be given access."
"We are delighted with the approval of ElapraseŽ in Europe, the first and only enzyme replacement treatment for Hunter syndrome. This decision reflects Shire's commitment not only to the development of ElapraseŽ, but also our wider commitment to developing innovative therapies for other, little researched genetic disorders", said Matthew Emmens, chief executive officer of Shire. "We believe the availability of ElapraseŽ for this rare but devastating condition will potentially have a huge impact on patients' lives. We remain committed to further research and monitoring of clinical outcomes in these patients to help them to maximise the benefits from this treatment".
Marketing authorisation for ElapraseŽ follows positive opinion issued by The Committee for Medicinal Products for Human Use (CHMP) in October 2006. The data supporting the licence has come from a comprehensive study which is the largest and longest registration study  of any lysosomal disorder. ElapraseŽ will be launched across Europe over the next 18 months.
 European Public Assessment Report for Elaprase (idursulfase) http://www.emea.europa.eu
 Froissart R et al. Mucopolysaccharidosis type II - genotype/phenotype aspects. Acta Paediatr suppl. 2002;439:82-87
 Kolodny EH et al. Storage diseases of the reticuloendothelial system. In: Nathan DG et al. Nathan and Oski's Hematology of Infancy and Childhood. 5th ed. Philadelphia, Pa: WB Saunders Co;1998:1461-1507
 Vellodi A et al. Long-term follow-up following bone marrow transplantation for Hunter Disease. J Inher Metab Dis, 1999:22:638-648
 Hunter syndrome. National Organization for Rare Disorders. www.rarediseases.org. January 2006
 Medline Plus. Hunter syndrome. http://www.nlm.nih.gov/medlineplus/ency/article/001203.htm. Accessed Nov 2006
 Muenzer J. et al. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccaridosis II (Hunter syndrome) Genet Med 2006 Aug;8(8):465-73
 American Thoracic Society. ATS Statement: Guidelines for the Six-Minute Walk Test Am J Respir Crit Care 2002:166:111-17
Notes to editors
Idursulfase is a purified form of the lysosomal enzyme iduronate-2-sulfatase and is produced by recombinant DNA technology in a human cell line.
About Hunter Outcome Survey
Shire 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 and the analysis and dissemination of this information will Allow for further understanding of Hunter syndrome and disease education on a global scale.
About Hunter Syndrome
Shire estimates that there are approximately 2,000 patients worldwide afflicted with Hunter Syndrome in countries where reimbursement may be possible.
Shire's strategic goal is to become the leading specialty pharmaceutical 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), SPD465 (extended release triple-bead mixed amphetamine salts) (ADHD), MESAVANCE (mesalamine) with MMX technology (SPD476) (ulcerative colitis) and VYVANSE(TM) (NRP104) (lisdexamfetamine dimesylate) (ADHD), including its scheduling classification by the Drug Enforcement Administration in the United States; Shire's ability to secure new products for commercialization and/or development; and other risks and uncertainties detailed from time to time in Shire's and its predecessor registrant Shire Pharmaceuticals Group 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, 2005.
Source: Shire PLC
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