, November 16, 2016
CUVITRU, now available in the U.S., expands Shire's immunoglobulin portfolio of treatment options to meet the unique needs of children and adults living with primary immunodeficiency
Shire plc (LSE: SHP, NASDAQ: SHPG) announces the launch of CUVITRU [Immune Globulin Subcutaneous (Human), 20% ution]?, the first and only Subcutaneous 20% treatment option without proline available in the U.S. to treat adult and pediatric patients (two years of age and older) with primary immunodeficiency (PI).
The U.S. Food and Drug Administration (FDA) approved CUVITRU in September 2016 as a subcutaneous immune globulin (IG) replacement therapy for PI, a group of more than 300 genetic disorders in which part of the body's immune system is missing or functions improperly, in some cases making it more difficult to fight off infections., PI affects up to six million people worldwide.,
People living with PI often face significant challenges relating to their disease and treatment. CUVITRU offers patients flexibility and control to manage their treatment in a way that best fits their needs and condition. In clinical trials, CUVITRU was infused weekly in under an hour in one to two sites for most patients. However, CUVITRU can be administered daily to biweekly and in up to four infusion sites, based on a patient's individual needs. CUVITRU is also the only 20% subcutaneous IG treatment option with the ability to infuse up to 60 mL (12 grams) per site and up to 60 mL per hour per site, as tolerated, allowing for fewer infusion sites and shorter infusion durations compared to other conventional subcutaneous IG treatments.
"We welcome CUVITRU as a new treatment option that will help patients and families manage their PI and adapt their treatment regimen to meet their personal needs," said Fred Modell, cofounder of the Jeffrey Modell Foundation, an organization devoted to early and precise diagnosis and care of PI patients. "We commend Shire's commitment to providing a range of treatment options that aim to improve the lives of PI patients."
Perry Sternberg, Head of U.S. Commercial, Shire, said: "CUVITRU offers an important new option for patients living with PI, providing the opportunity for fewer infusion sites and shorter infusion durations. Shire has focused on making CUVITRU available quickly following FDA approval. We recognize the importance of ensuring patients have access to a broad portfolio of IG treatment options so they can choose a therapy that best fits their needs."
The FDA approval was based on positive results from two prospective open-label, non-controlled, multi-center Phase II/III studies assessing the efficacy, tolerability and pharmacokinetics of CUVITRU in patients two years and older with PI. In the North American Clinical Study nearly all infusions (99.8%) were completed without a reduction, interruption, or discontinuation for tolerability reasons and 84.9% of infusions were administered using = 2 infusion sites. Regardless of infusion rate or volume per site, CUVITRU was generally associated with a low incidence of local adverse and systemic reactions (0.022/infusion and 0.042/infusion, respectively).
With the availability of CUVITRU, Shire expands its industry-leading portfolio of intravenous and subcutaneous IG products available for PI patients. In June 2016, Shire announced the successful completion of a decentralized procedure to support approval by 17 authorities in Europe for CUVITRU. The company expects to initiate additional global regulatory submissions for CUVITRU in late 2016 and 2017.
For more information on CUVITRU, please visit http://www.cuvitru.com.
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 inherited and 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.
About CUVITRU [Immune Globulin Subcutaneous (Human), 20% Solution]
CUVITRU is an Immune Globulin Subcutaneous (Human) (IGSC), 20% Solution indicated as replacement therapy for primary humoral immunodeficiency (PI) in adult and pediatric patients two years of age and older.
CUVITRU is for subcutaneous infusion only.
Detailed Important Risk Information
BOXED WARNING: THROMBOSIS
Thrombosis may occur with immune globulin products, including CUVITRU. Risk factors may include: advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling vascular catheters, hyperviscosity and cardiovascular risk factors.
For patients at risk of thrombosis, administer CUVITRU at the minimum dose and infusion rate practicable. Ensure adequate hydration in patients before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk of hyperviscosity.
CUVITRU is contraindicated in patients who have had an anaphylactic or severe systemic hypersensitivity reaction to the subcutaneous administration of human immune globulin and in IgA-deficient patients with antibodies against IgA and a history of hypersensitivity to human immune globulin treatment.
WARNINGS and PRECAUTIONS
Hypersensitivity: Severe hypersensitivity reactions may occur, even in patients who have tolerated previous treatment with human immune globulin. IgA-deficient patients with antibodies to IgA are at greater risk of developing potentially severe hypersensitivity and anaphylactic reactions.
Renal Dysfunction/Failure: Monitor renal function and urine output and consider lower, more frequent dosing in patients who are at risk of developing renal dysfunction because of pre-existing renal insufficiency or predisposition to acute renal failure.
Thrombosis: Monitor for signs and symptoms of thrombosis and assess blood viscosity for those at risk for hyperviscosity.
Aseptic Meningitis Syndrome (AMS): Monitor for clinical signs and symptoms of AMS.
Hemolysis: Monitor for clinical signs and symptoms of hemolysis and delayed hemolytic anemia.
Transfusion-Related Acute Lung Injury (TRALI): Monitor for pulmonary adverse reactions associated with TRALI.
Transmittable Infectious Agents: Because CUVITRU is made from human plasma, it may carry a risk of transmitting infectious agents, such as viruses and other pathogens. No confirmed cases of transmission of viral diseases or variant Creutzfeldt-Jakob disease (vCJD) have been associated with CUVITRU.
Interference with Laboratory Tests: False positive serological test results, with the potential for misleading interpretation, may occur as the result of passively transferred antibodies.
The most common adverse reactions observed in clinical trials in = 5% of patients were: local adverse reactions, systemic adverse reactions including headache, nausea, fatigue, diarrhea, and vomiting.
Please see Full Prescribing Information, including Boxed Warning regarding Thrombosis, available at: http://www.shirecontent.com/PI/PDFS/Cuvitru_USA_ENG.pdf.
SHIRE and the Shire Logo are registered trademarks of Shire Pharmaceutical Holdings Ireland Limited or its affiliates. CUVITRU is a trademark or registered trademark of Baxalta Incorporated, a wholly owned, indirect subsidiary of Shire plc.
1. Bousfiha AA et al. Primary immunodeficiency diseases worldwide: more common than generally thought. J Clin Immunol. 2013 Jan;33(1):1-7.
2. Picard C, Al-Herz W, et al. Primary Immunodeficiency Diseases: an Update on the Classification from the International Union of Immunological Societies Expert Committee for Primary Immunodeficiency. J Clin Immunol. 2015 Nov;35(8):696-726.
3. IDF Patient & Family Handbook for Primary Immunodeficiency Diseases. 5th edition. Blaese. 2013.
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:
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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.
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