Shire Launches Pediatric Indication for Immunodeficiency Treatment HyQvia in Europe

ZUG, Switzerland, July 21, 2016 /PRNewswire/ --

HyQvia (Human Normal Immunoglobulin 10% and Recombinant Human Hyaluronidase) is the only subcutaneous IG treatment for primary and certain secondary immunodeficiencies that can be administered in one site, once a month (up to every four weeks) 

Shire plc (LSE: SHP, NASDAQ: SHPG) is launching a pediatric indication for HyQvia (Human Normal Immunoglobulin (10%), Recombinant Human Hyaluronidase) across Europe. This follows the recent marketing authorisation granted by the European Commission to Baxalta, now part of Shire, in June 2016.

HyQvia is a replacement therapy to treat primary and certain secondary immunodeficiencies, a group of disorders in which part of the body's immune system is missing or does not function properly, in some cases making it more difficult to fight off infections. It is estimated that as many as six million children and adults may be affected by Primary Immunodeficiencies (PI) worldwide.

Shire is commercially introducing the new indication across the member states of Europe, starting this month with Germany, The Netherlands, Ireland, Greece, Slovakia, Denmark, Sweden and Norway.

"We are pleased to bring pediatric patients a new therapeutic option as we build on our broad immunoglobulin (IG) portfolio for patients with immune deficiencies," said Ueli Fankhauser, Head, Global Product Strategy. "We intend to expand the availability of HyQvia to more patients in additional geographies, with the goal of reducing the treatment burden for patients worldwide."

HyQvia was developed with a focus on addressing unmet treatment needs for patients. The treatment, which does not include proline, offers a demonstrated safety and efficacy profile (based on the pivotal study among 87 patients) as a two-part infusion combining Halozyme's recombinant human hyaluronidase with immunoglobulin (Ig). HyQvia delivers a full dose of treatment in 1-2 infusion sites, up to once a month (up to every four weeks) for many patients, and can be self-administered at home after appropriate training. The flexibility of the treatment approach was noted by patient feedback during the pivotal study, in which 79 percent of patients and their caregivers (those <18) preferred to continue treatment with HyQvia rather than an intravenous or conventional subcutaneous treatment.

Shire (Baxter at the time) initially obtained marketing authorization for HyQvia in Europe in May 2013. The product is also licensed in the US, in Puerto Rico, and Australia, and currently under review in Brazil, Columbia, Mexico, and Argentina.

About HyQvia (Human Normal Immunoglobulin (10%), Recombinant Human Hyaluronidase) in Europe  

Indication and Usage 

In Europe today, HyQvia is indicated as replacement therapy in all patients in primary immunodeficiency syndromes with impaired antibody production, hypogammaglobulinaemia and recurrent bacterial infections in patients with chronic lymphocytic leukaemia (CLL), in whom prophylactic antibiotics have failed or are contra-indicated, hypogammaglobulinaemia and recurrent bacterial infections in multiple myeloma (MM) patients, hypogammaglobulinaemia in patients pre- and post-allogeneic hematopoietic stem cell transplantation (HSCT).

For more information on HyQvia in Europe, please visit http://www.ema.europa.eu.

Important Risk Information 

HyQvia must not be used by patients with a hypersensitivity to human immunoglobulins, especially in very rare cases of IgA deficiency when the patient has antibodies against IgA. HyQvia must not be used by patients with a systemic hypersensitivity to hyaluronidase or recombinant human hyaluronidase. HyQvia must not be used by patients with a hypersensitivity to any of the excipients, including glycine.

HyQvia must not be given intravenously.

Patients should be closely monitored and carefully observed for any adverse reactions throughout the infusion period, particularly patients starting with HyQvia treatment. In case of adverse reaction, either the rate of administration must be reduced or the infusion stopped. The treatment required depends on the nature and severity of the adverse reaction.

In case of shock, standard medical treatment for shock should be implemented.

Thromboembolic events (e.g. myocardial infarction, cerebral vascular accident, deep vein thrombosis, and pulmonary embolism), renal dysfunction/failure, aseptic meningitis syndrome, and hemolysis have been observed with IG 10% administered intravenously and cannot be excluded with use of HyQvia. Thrombotic events and haemolysis have also been reported in association with the subcutaneous administration of immunoglobulin products.

Human normal immunoglobulin and human serum albumin (stabilizer of the recombinant human hyaluronidase) are produced from human plasma. Standard measures to prevent infections resulting from the use of medicinal products prepared from human blood or plasma include selection of donors, screening of individual donations and plasma pools for specific markers of infection and the inclusion of effective manufacturing steps for the inactivation/removal of viruses. Despite this, when medicinal products prepared from human blood or plasma are administered, the possibility of transmitting infectious agents cannot be totally excluded. This also applies to unknown or emerging viruses and other pathogens.

About Primary Immunodeficiency 

Primary immunodeficiencies (PI) are a group of more than 300 disorders in which part of the body's immune system is missing or does not function properly. Normally, the immune system protects the body from pathogenic microorganisms like bacteria, viruses, and fungi, which can cause infectious diseases. When any part of a person's immune system is absent or dysfunctional, the individuals are susceptible to infections, and it may take longer to recover from infections. When a defect in the immune system is genetically determined, it is called primary immune deficiency. It is estimated that as many as six million children and adults may be affected by PI worldwide.

NOTES TO EDITORS 

Shire is the leading global biotechnology company focused on serving people with rare diseases and other highly specialized conditions. We strive to develop best-in-class products, many of which are available in more than 100 countries, across core therapeutic areas including Hematology, Immunology, Neuroscience, Ophthalmics, Lysosomal Storage Disorders, Gastrointestinal / Internal Medicine / Endocrine and Hereditary Angioedema; and a growing franchise in Oncology.

Our employees come to work every day with a shared mission: to develop and deliver breakthrough therapies for the hundreds of millions of people in the world affected by rare diseases and other high-need conditions, and who lack effective therapies to live their lives to the fullest.

http://www.shire.com

Forward-Looking Statements 

Statements included herein that are not historical facts, including without limitation statements concerning future strategy, plans, objectives, expectations and intentions, the anticipated timing of clinical trials and approvals for, and the commercial potential of, inline or pipeline products 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 adversely affected. The risks and uncertainties include, but are not limited to, the following:

• disruption from the acquisition and integration of Baxalta Incorporated ("Baxalta") may make it more difficult to conduct business as usual or maintain relationships with patients, physicians, employees or suppliers;
• the company may not achieve some or all of the anticipated benefits of Baxalta's spin-off from Baxter International, Inc. ("Baxter") and the acquisition may have an adverse impact on Baxalta's existing arrangements with Baxter, including those related to transition, manufacturing and supply services and tax matters;
• the failure to achieve the strategic objectives with respect to the acquisition of Baxalta may adversely affect the company's financial condition and results of operations;
• products and product candidates may not achieve commercial success;
• product sales from ADDERALL XR and INTUNIV are subject to generic competition;
• the failure to obtain and maintain reimbursement, or an adequate level of reimbursement, by third-party payers in a timely manner for the company's products may affect future revenues, financial condition and results of operations, particularly if there is pressure on pricing of products to treat rare diseases;
• supply chain or manufacturing disruptions may result in declines in revenue for affected products and commercial traction from competitors; regulatory actions associated with product approvals or changes to manufacturing sites, ingredients or manufacturing processes could lead to significant delays, an increase in operating costs, lost product sales, an interruption of research activities or the delay of new product launches;
• the successful development of products in various stages of research and development is highly uncertain and requires significant expenditures and time, and there is no guarantee that these products will receive regulatory approval;
• the actions of certain customers could affect the company's ability to sell or market products profitably, and fluctuations in buying or distribution patterns by such customers can adversely affect the company's revenues, financial condition or results of operations;
• investigations or enforcement action by regulatory authorities or law enforcement agencies relating to the company's activities in the highly regulated markets in which it operates may result in significant legal costs and the payment of substantial compensation or fines;
• adverse outcomes in legal matters, tax audits and other disputes, including the company's ability to enforce and defend patents and other intellectual property rights required for its business, could have a material adverse effect on the company's revenues, financial condition or results of operations;
• Shire is undergoing a corporate reorganization and was the subject of an unsuccessful acquisition proposal and the consequent uncertainty could adversely affect the company's ability to attract and/or retain the highly skilled personnel needed to meet its strategic objectives;
• failure to achieve the strategic objectives with respect to Shire's acquisition of NPS Pharmaceuticals Inc. or Dyax Corp. ("Dyax") may adversely affect the company's financial condition and results of operations;
• the company is dependent on information technology and its systems and infrastructure face certain risks, including from service disruptions, the loss of sensitive or confidential information, cyber-attacks and other security breaches or data leakages that could have a material adverse effect on the company's revenues, financial condition or results of operations;
• the company may be unable to retain and hire key personnel and/or maintain its relationships with customers, suppliers and other business partners;
• difficulties in integrating Dyax or Baxalta into Shire may lead to the company not being able to realize the expected operating efficiencies, cost savings, revenue enhancements, synergies or other benefits at the time anticipated or at all; and

other risks and uncertainties detailed from time to time in Shire's,  Dyax's or Baxalta's filings with the Securities and Exchange Commission, including those risks outlined in "ITEM 1A: Risk Factors" in Shire's and Baxalta's Annual Reports on Form 10-K for the year ended December 31, 2015.

All forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof. Except to the extent otherwise required by applicable law, we do not undertake any obligation to republish revised forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events.

For further information please contact:  

Investor Relations 
Sarah Elton-Farr
seltonfarr@shire.com
+44 1256 894157

Ian Karp
ikarp@shire.com
+1-781-482-9018

Robert Coates
rcoates@shire.com
+44-1256-894874

Media 
Gwen Fisher
gfisher@shire.com
+1-484-595-9836

Debbi Ford
debbi.ford@shire.com  
+1-224-727-2079