24th Jul 2006 17:58
Shire's ELAPRASE¢â€ž¢ (idursulfase) Approved by the Food and Drug Administration(FDA) for Hunter SyndromeBasingstoke, UK and Philadelphia, US - July 24, 2006 - Shire plc (LSE: SHP,NASDAQ: SHPGY, TSX: SHQ) today announced that the FDA has granted marketingapproval for ELAPRASE, a human enzyme replacement therapy for the treatment ofHunter syndrome, also known as Mucopolysaccharidosis II (MPS II). Huntersyndrome is a rare, life-threatening genetic condition that results from theabsence 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 is the first and only treatment approved for people suffering fromHunter syndrome. The product, which is given as weekly infusions, replaces themissing enzyme that Hunter syndrome patients fail to produce in sufficientquantities.Shire expects to launch ELAPRASE in the United States within the next 30 days."The FDA approval of ELAPRASE marks a significant milestone in Shire's effortto provide meaningful treatments for patients suffering from genetic diseases,"said Matthew Emmens, chief executive officer of Shire. "A hallmark of theELAPRASE program is the commitment demonstrated by patients and families,investigators and Shire employees involved in the development effort. We lookforward to making ELAPRASE available to patients in the coming weeks.""Regulatory approval of ELAPRASE will enable physicians to move needy patientsbeyond palliative care and make Hunter syndrome a treatable disease," saidJoseph Muenzer, MD., Ph.D, of the University of North Carolina at Chapel Hill."Until today, there were no options for addressing the underlying cause of thisdevastating disease."Shire submitted a Marketing Authorization Application (MAA) for ELAPRASE to theEuropean Medicines Agency (EMEA) on December 1, 2005. Based on averageevaluation times, Shire anticipates completion of the EMEA review by year end.In European countries that have mechanisms for pre-approval access, Shire hasalso submitted applications.Clinical Trial ResultsA 53-week, randomized, double-blind, placebo-controlled Phase II/III trialdemonstrated that ELAPRASE provides clinically important benefits to Huntersyndrome patients. The primary efficacy endpoint of the trial was a compositeanalysis of changes from baseline in two clinical measures: a 6-minute walktest and percent predicted forced vital capacity. Shire is pleased to reportthat this endpoint achieved statistical significance compared to placebo. Afterone year of treatment, patients receiving weekly infusions of ELAPRASEexperienced a mean increase in the distance walked in six minutes of 35 meterscompared to patients receiving placebo.Safety DataTreatment with ELAPRASE was generally well-tolerated by patients in the PhaseII/III trial. Adverse reactions were commonly reported in association withinfusions, and were generally mild to moderate.The ELAPRASE label includes a boxed warning with information on the potentialfor hypersensitivity reactions. The boxed warning states that "Anaphylactoidreactions, which may be life threatening, have been observed in some patientsduring ELAPRASE infusions. Therefore, appropriate medical support should bereadily available when ELAPRASE is administered. Patients with compromisedrespiratory function or acute respiratory disease may be at risk of seriousacute exacerbation of their respiratory compromise due to infusion reactions,and require additional monitoring."In all phases of clinical study for ELAPRASE, 11 patients experiencedsignificant hypersensitivity reactions during 19 of 8,274 infusions (0.2%) andno patients discontinued treatment permanently as a result of ahypersensitivity reaction. The most common adverse events observed in >30% ofpatients during the Phase II/III trial were pyrexia, headache and arthralgia.Fifty-one percent (32 of 63) of patients in the weekly ELAPRASE treatment armin the pivotal clinical study (53-week placebo-controlled study with anopen-label extension) developed anti-idursulfase IgG antibodies.About ELAPRASEELAPRASE is a purified form of the lysosomal enzyme iduronate-2-sulfatase andis produced by recombinant DNA technology in a human cell line.In conjunction with the market approval of ELAPRASE, Shire Human GeneticTherapies (the Shire business unit focused on genetic diseases) has introduceda new product support center called OnePathSM for the U.S market. OnePathSM isa single source of product support for healthcare providers, patients and theirfamilies, where personalized, comprehensive information about ELAPRASE isavailable from a case manager. Case managers can provide information aboutcoding, reimbursement and insurance verification, authorization letters,product access and treatment center locations. OnePathSM also offers educationabout Hunter syndrome and can refer patients to additional support services, ifneeded.Shire Human Genetic Therapies is actively tracking health data amongindividuals affected by Hunter syndrome as part of the company's long-termoutcome survey, called the Hunter Outcome Survey (HOS). HOS is designed tosupport the gathering, analysis, reporting and sharing of data from around theworld about Hunter syndrome. Shire believes that the inclusion of all peopleaffected by Hunter syndrome and the analysis and dissemination of thisinformation will allow for further understanding of Hunter syndrome and diseaseeducation on a global scale.More information about ELAPRASE and Hunter syndrome is available at http://www.elaprase.com, www.hunterpatients.com, or through OnePath SM at 1 (866)888-0660.About Hunter SyndromeHunter syndrome (MPS II) is a serious genetic disorder mainly affecting malesthat interferes with the body's ability to break down and recycle wastesubstances called mucopolysaccharides, also known as glycosaminoglycans or GAG.Hunter syndrome is one of several related lysosomal storage diseases.In Hunter syndrome, cumulative buildup of GAG in cells throughout the bodyinterferes with the way certain tissues and organs function, leading to severeclinical complications and early mortality. Physical manifestations for somepeople with Hunter syndrome may include distinct facial features, a large headand an enlarged abdomen. People with Hunter syndrome may also experiencehearing loss, thickening of the heart valves leading to a decline in cardiacfunction, obstructive airway disease, sleep apnea, and enlargement of the liverand spleen. In some cases, central nervous system involvement leads toprogressive neurologic decline.Shire estimates that there are approximately 2,000 patients worldwide afflictedwith Hunter syndrome in areas where reimbursement may be possible. Shiresestimates that the U.S. accounts for approximately 25% of the global market forHunter syndrome.For further information please contact:Investor Relations Clƒ©a Rosenfeld (Rest of the World) +44 1256 894 160 Brian Piper (North America) +1 484 595 8252 Media Jessica Mann (Rest of the World) +44 1256 894 280 Matthew Cabrey (North America) +1 484 595 8248 Notes to editorsSHIRE PLCShire's strategic goal is to become the leading specialty pharmaceuticalcompany that focuses on meeting the needs of the specialist physician. Shirefocuses its business on attention deficit and hyperactivity disorder,gastrointestinal, renal diseases and human genetic therapies. The structure issufficiently flexible to allow Shire to target new therapeutic areas to theextent opportunities arise through acquisitions. Shire believes that acarefully selected portfolio of products with a strategically aligned andrelatively small-scale sales force will deliver strong results.Shire's focused strategy is to develop and market products for specialtyphysicians. Shire's in-licensing, merger and acquisition efforts are focused onproducts in niche markets with strong intellectual property protection eitherin 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 OF1995Statements included herein that are not historical facts are forwarding-lookingstatements. Such forward-looking statements involve a number of risks anduncertainties and are subject to change at any time. In the event such risks oruncertainties materialize, Shire plc's results could be materially affected.The risks and uncertainties include, but are not limited to: risks associatedwith 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 plc's AttentionDeficit and Hyperactivity Disorder ("ADHD") franchise; patents, including butnot limited to, legal challenges relating to Shire plc's ADHD franchise;government regulation and approval, including but not limited to the expectedproduct approval dates of SPD503 (ADHD), SPD465 (ADHD), MESAVANCE TM (SPD476)(ulcerative colitis) and NRP104 (ADHD), including its scheduling classificationby the Drug Enforcement Administration in the United States; Shire plc'sability to benefit from the acquisition of Transkaryotic Therapies Inc.; Shireplc's ability to secure new products for commercialization and/or development;and other risks and uncertainties detailed from time to time in Shire plc's andits predecessor registrant Shire Pharmaceuticals Group plc's filings with theUS Securities and Exchange Commission, including Shire plc's Annual Report onForm 10-K for the year ended December 31, 2005. Hampshire International Business Park Chineham Basingstoke Hampshire RG24 8EP United Kingdom Tel +44 (0)1256 894000 Fax +44 (0)1256 894708 www.shire.com Press Release Registered in England 2883758 Registered Office as aboveENDSHIRE PLCRelated Shares:
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