Zug, Switzerland. – September 23, 2016 – Shire plc (LSE: SHP, NASDAQ: SHPG) will showcase data across its global leading immunoglobulin (IG) portfolio during the 17th Biennial Meeting of the European Society for Immunodeficiencies (ESID), taking place September 21-24, 2016 in Barcelona.
Presentations will include results of studies examining the safety, efficacy and tolerability profiles of Shire’s IG treatment options for immune deficiencies, including primary immunodeficiency (PI). Shire will complement the data on treatment options with research on the burden PI patients experience relating to their disease and treatment, as part of Shire’s commitment to delivering advanced, individualized treatment options for people with PI.
“ESID is an important opportunity for the scientific community to come together and reaffirm our shared commitment to advancing treatment options for people with immune deficiencies,” said Philip J. Vickers, Ph.D., Head of Research and Development, Shire. “Shire is proud to be a leading voice in this conversation. We remain dedicated to understanding the patient journey and to driving innovation that addresses the evolving needs of patients.”
Highlights of Shire’s Presentations at ESID
Efficacy, Safety/Tolerability, and Pharmacokinetics of Human Immune Globulin Subcutaneous, 20%: Final Analysis of a Phase 2/3 Study in Patients with Primary Immunodeficiency Disease in North America
This data, published in the August issue of the Journal of Clinical Immunology, highlights final results from a Phase 2/3 study of Cuvitru, 20% IGSC. The prospective, open-label, non-controlled, multi-center study was conducted to assess the efficacy, safety, and pharmacokinetics (PK) of 20% IGSC among 74 North American patients at least two years old with PI. Data from a parallel study among patients in Europe was published in the September issue of Clinical and Experimental Immunology.
Cuvitru [Immune Globulin Subcutaneous (Human) 20% Solution], a treatment without proline for pediatric and adult patients with primary and certain secondary immunodeficiency disorders, is the latest treatment in Shire’s broad IG portfolio. In June 2016, Shire announced the successful completion of a decentralized procedure to support approval by 17 authorities in Europe for Cuvitru; the treatment was then approved in the United States earlier in September 2016.
Long-Term Safety, Efficacy, and Tolerability of Recombinant Human Hyaluronidase-Facilitated Subcutaneous Infusion of Immunoglobulin in Patients Aged <18 Years with Primary Immunodeficiency (PIDD)
This data evaluates the efficacy, safety and tolerability of HyQvia (Human Normal Immunoglobulin 10% and Recombinant Human Hyaluronidase) among patients 18 years of age and younger treated in HyQvia’s pivotal phase 3 study and its extension study. HyQvia is currently available for adults with PI in the United States and in 14 EU member states, where it is indicated for PI and certain secondary immunodeficiency disorders. In June 2016, Shire received the European Commission Decision for approval for the pediatric indication for HyQvia.
Interim Results of a Non-Interventional Post-Authorization Safety Study (PASS) on the Long-Term Safety of IGSC 10% Infusion Facilitated With Recombinant Human Hyaluronidase
This post-authorization safety study (PASS) was initiated in Europe in July 2014 to acquire additional safety data on HyQvia. The ongoing prospective, non-interventional, open label, uncontrolled, multicentre PASS is designed to evaluate the long-term local and systemic effects of HyQvia, as well as prescribed treatment regimens and treatment administration in routine clinical practice, in adults with PI, myeloma and chronic lymphocytic leukemia.
Understanding the Burden of Receiving Immunoglobulin Treatment for Primary Immunodeficiency Disorders: A Qualitative Study
People living with PI often face significant challenges relating to their disease and treatment. This qualitative research aims to better understand the burden of IG treatment in order to identify strategies to improve the lives of PI patients and their caregivers by tailoring treatment plans to individual needs. Researchers interviewed a diverse group of 52 PI patients receiving a range of type and mode of IG treatments via a purposive sampling method to learn more about their perspectives on the treatment experience.
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 Cuvitru 200 mg/ml solution for subcutaneous injection in Europe
After marketing authorization, Cuvitru 200 mg/ml solution for subcutaneous injection will be indicated as a subcutaneous administration (SCIg) replacement therapy in adults, and children and adolescents (0 - 18 years) 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 haematopoietic stem cell transplantation (HSCT).
Detailed Important Risk Information
Hypersensitivity to the active substance or to any of the excipients.
Severe IgA deficiency and a history of hypersensitivity to human immunoglobulin treatment.
Cuvitru must not be given intravascularly or intramuscularly.
WARNINGS and PRECAUTIONS
If Cuvitru is accidentally administered into a blood vessel patients could develop shock.
The recommended infusion rate must be closely followed. Patients must be closely monitored and carefully observed for any symptoms throughout the infusion period.
Certain adverse reactions may occur more frequently in patients who receive human normal immunoglobulin for the first time or, in rare cases, when the human normal immunoglobulin product is switched or when there has been a long interval since the previous infusion.
Potential complications can often be avoided by:
- Initially injecting the product slowly
- Ensuring that patients are carefully monitored for any symptoms throughout the infusion period. In particular, patients naive to human normal immunoglobulin, patients switched from an alternative immunoglobulin product or when there has been a long interval since the previous infusion should be monitored during the first infusion and for the first hour after the first infusion, in order to detect potential adverse signs.
All other patients should be observed for at least 20 minutes after administration.
In case of adverse reaction, either the rate of administration must be reduced or the infusion stopped. Suspicion of severe hypersensitivity or anaphylactic-type reactions requires immediate discontinuation of the injection. 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, hemolysis and interference with serological testing have been observed with IG administered intravenously and cannot be excluded with use of Cuvitru. Thrombotic events and hemolysis have also been reported in association with the subcutaneous administration of immunoglobulin products.
Human normal immunoglobulin is produced from human plasma and may carry a risk of transmitting infectious agents.
© 2016 Shire US Inc., Lexington, MA 02421. All rights reserved. 1-800-828-2088.
SHIRE and the Shire Logo are registered trademarks of Shire Pharmaceutical Holdings Ireland Limited or its affiliates.
Baxalta, HyQvia and Cuvitru are registered trademarks of Baxalta Incorporated, a wholly owned, indirect subsidiary of Shire plc.
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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.
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:
- Shire’s products may not be a commercial success;
- increased pricing pressures and limits on patient access as a result of governmental regulations and market developments may affect Shire’s future revenues, financial condition and results of operations;
- Shire conducts its own manufacturing operations for certain of its products and is reliant on third party contract manufacturers to manufacture other products and to provide goods and services. Some of Shire’s products or ingredients are only available from a single approved source for manufacture. Any disruption to the supply chain for any of Shire’s products may result in Shire being unable to continue marketing or developing a product or may result in Shire being unable to do so on a commercially viable basis for some period of time;
- the manufacture of Shire’s products is subject to extensive oversight by various regulatory agencies. Regulatory approvals or interventions associated with 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;
- certain of Shire’s therapies involve lengthy and complex processes, which may prevent Shire from timely responding to market forces and effectively managing its production capacity;
- Shire has a portfolio of products in various stages of research and development. The successful development of these products 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 Shire’s ability to sell or market products profitably. Fluctuations in buying or distribution patterns by such customers can adversely affect Shire’s revenues, financial conditions or results of operations;
- Shire’s products and product candidates face substantial competition in the product markets in which it operates, including competition from generics;
- adverse outcomes in legal matters, tax audits and other disputes, including Shire’s ability to enforce and defend patents and other intellectual property rights required for its business, could have a material adverse effect on the combined company’s revenues, financial condition or results of operations;
- inability to successfully compete for highly qualified personnel from other companies and organizations;
- failure to achieve the strategic objectives with respect to Shire’s acquisition of NPS Pharmaceuticals, Inc., Dyax Corp. (“Dyax”) or Baxalta Inc. (“Baxalta”)may adversely affect Shire’s financial condition and results of operations;
- Shire’s growth strategy depends in part upon its ability to expand its product portfolio through external collaborations, which, if unsuccessful, may adversely affect the development and sale of its products;
- a slowdown of global economic growth, or economic instability of countries in which Shire does business, as well as changes in foreign currency exchange rates and interest rates, that adversely impact the availability and cost of credit and customer purchasing and payment patterns, including the collectability of customer accounts receivable;
- failure of a marketed product to work effectively or if such a product is the cause of adverse side effects could result in damage to the Shire’s reputation, the withdrawal of the product and legal action against Shire;
- investigations or enforcement action by regulatory authorities or law enforcement agencies relating to Shire’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;
- Shire 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 Shire’s revenues, financial condition or results of operations;
- Shire incurred substantial additional indebtedness to finance the Baxalta acquisition, which may decrease its business flexibility and increase borrowing costs;
- difficulties in integrating Dyax or Baxalta into Shire may lead to the combined 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 filings with the Securities and Exchange Commission, including those risks outlined in “ITEM 1A: Risk Factors” in Shire’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2016.
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 update or revise forward-looking statements, whether as a result of new information, future events or otherwise.