LEXINGTON, Massachusetts,
November 12, 2016 /PRNewswire/ --
Data reveals key findings
on the tolerability of CUVITRU [Immune
Globulin Subcutaneous (Human), 20% Solution] for
patients, regardless of treatment infusion
volume and rates
Shire plc (LSE: SHP, NASDAQ: SHPG) will present additional data
supporting the tolerability of CUVITRU [Immune Globulin
Subcutaneous (Human), 20% Solution] in patients two years of age
and older with primary immunodeficiency (PI) diseases in
North America at the 2016 Annual
Scientific Meeting of the American College of Allergy, Asthma &
Immunology in San Francisco. The
presentation will highlight key findings on established
tolerability of CUVITRU for PI patients who received CUVITRU in the
Phase II/III North American clinical trial. Of note, increased
infusion volumes and rates were not associated with an increase in
causally-related local adverse reactions, and infusions were
well-tolerated during onboarding and throughout the study.
"As the latest treatment to be added to Shire's industry-leading
IG portfolio, CUVITRU exemplifies our commitment to reducing the
burden of PI for patients living with this challenging, often
debilitating condition," said Philip J.
Vickers, Ph.D., Head of Research and Development, Shire. "As
a customizable therapy, CUVITRU offers patients and physicians
multiple options to tailor their IG treatment to fit their needs
and preferences, such as varying the volume/site, the dosing
frequency, and infusion rate."
PI is 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.[1],[2]
PI affects six million people worldwide, of which approximately
250,000 reside in the
U.S.[3],[4]
Most people with PI have abnormally low or nonexistent IG levels,
and may benefit from IG replacement treatment to help the body
prevent infections. Since it only offers temporary protection, many
people with PI require IG replacement treatment throughout their
lives.[5]
"ACAAI's annual meeting is a welcome opportunity for the
immunology community to learn more about the benefits CUVITRU can
offer patients with PI looking to take control of their IG
treatment," said Sudhir Gupta, MD,
PH.D., MACP, Chief of Basic and Clinical Immunology, and Director,
Programs in Primary Immunodeficiency and Aging, at University of California, Irvine. "The data
reinforces that patients can work with their physician to
individualize their dosage and administration schedule, allowing
for greater flexibility and control within their normal daily
routine."
As demonstrated in the clinical trials, CUVITRU offers patients
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. The data to be presented at ACAAI
offers additional evidence that patients achieved the increased
rate and volume, without compromising tolerability. Approximately
72% of patients in the clinical trial achieved the maximum infusion
rate of 60 mL per hour per site, occurring after a median time of 3
infusions with no association between infusion rate and causally
related local adverse event (AE) rates. Importantly, 99.8%
infusions of CUVITRU were completed without a rate reduction,
interruption, or discontinuation indicating the infusions were well
tolerated. In addition, 75% of infusions delivered a volume of 30
mL per site or greater, with no association between volume per site
and causally-related local AEs.[6]
The full clinical trial results can be found in Journal of
Clinical Immunology.
CUVITRU is the latest product in Shire's industry-leading IG
portfolio. The U.S. Food and Drug Administration approved CUVITRU
in September 2016. Shire also
received successful completion of a decentralized procedure to
support CUVITRU approval by 17 authorities in Europe in June
2016. 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.[1]
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.[2]
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.
CONTRAINDICATIONS
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.
ADVERSE REACTIONS
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.
NOTES TO EDITORS
About Shire
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:
- 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.
References
- 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.
- IDF Patient & Family Handbook for Primary Immunodeficiency
Diseases. 5th edition. Blaese. 2013.
- Bousfiha AA et al. Primary immunodeficiency diseases worldwide:
more common than generally thought. J Clin Immunol. 2013
Jan;33(1):1-7.
- Immune Deficiency Foundation. Primary immune deficiency
diseases in America: 2007. The third national survey of patients.
May 2009.
- Immune Deficiency Foundation, "Immunoglobulin Therapy &
Other Medical Therapies for Antibody Deficiencies:"
http://primaryimmune.org/treatment-information/immunoglobulin-therapy/
. October 19, 2015.
- Gupta S. "Tolerability of the New Human Immune Globulin
Subcutaneous, 20% Preparation in Primary Immunodeficiency Diseases"
Annual Scientific Meeting of the American College of Allergy,
Asthma & Immunology, San
Francisco. November 10-14,
2016.
For further information please
contact:
Investor Relations
Sarah Elton-Farr seltonfarr@shire.com +44(0)1256-894157
Ian Karp ikarp@shire.com +1-781-482-9018
Robert Coates rcoates@shire.com +44(0)1256-894874
Media
Gwen Fisher gfisher@shire.com +1-484-595-9836
Debbi Ford debbi.ford@shire.com +1-617-949-9083
SOURCE Shire plc
