Shire Announces
FDA Approval for Label Expansion of
CINRYZE® (C1 esterase
inhibitor [human]) for Prevention of Attacks in Pediatric
Hereditary Angioedema Patients
-
CINRYZE is the first and only
therapy indicated in the U.S. to help prevent angioedema attacks in
pediatric patients with hereditary angioedema (HAE) as young as
6
-
Shire is the only drug
developer to complete a pediatric study for the prophylactic
treatment of HAE
-
HAE is a rare, genetic disorder
that causes debilitating, painful and sometimes life-threatening
swelling in the body
Dublin, Ireland - Thursday, June
21, 2018 - Shire plc (LSE: SHP, NASDAQ: SHPG), the leading
global biotechnology company focused on rare diseases, today
announced that the U.S. Food and Drug Administration (FDA) has
approved a label expansion for CINRYZE® (C1 esterase
inhibitor [human]), making it available to help prevent angioedema
attacks in children aged 6 years and older with hereditary
angioedema (HAE). CINRYZE has been approved in the U.S. since
October 2008 for routine prophylaxis against attacks in adolescents
and adults living with HAE.[1]
HAE is a rare, genetic disorder estimated to
affect about 1 in 10,000 to 1 in 50,000 people
worldwide.[2],[3] The
condition results in recurring attacks of edema (swelling) in
various parts of the body, including the abdomen, face, feet,
genitals, hands and throat that can be can debilitating and
painful.2,[4],[5]
Attacks that obstruct the airways (laryngeal attacks) are
potentially life-threatening due to the risk of
asphyxiation.2,4,5
Andreas Busch, Ph.D., Executive Vice President,
Head of Research and Development at Shire said: "Symptoms of HAE
often present in childhood with the average child experiencing
their first HAE attack around the age of 10.[6] With
the FDA label expansion of CINRYZE, children as young as 6 years
old living with HAE now have the first FDA approved treatment
option available to help prevent attacks."
The approval was based on data from a dedicated
Phase 3 multicenter single-blind study (0624-301) that evaluated
the use of CINRYZE in 12 patients living with HAE aged 7 to 11.
Compared to the baseline observational period, the mean reduction
in the normalized number of attacks for CINRYZE 500 U and CINRYZE
1,000 U was 71.1% and 84.5%, respectively. Both doses lessened the
severity of attacks and reduced the use of acute treatment compared
to baseline. The adverse reactions were headache, nausea, pyrexia
(fever), and infusion site erythema (redness of the skin). None of
these adverse reactions were severe, and none led to
discontinuation (n=12, ages 7-11).1
"This news is exciting for the HAE community
because those living with HAE who are as young as 6 have a new
option to help prevent attacks," said Anthony Castaldo, President
of the U.S. Hereditary Angioedema Association.
In March 2017, CINRYZE was granted European
Commission (EC) approval for the label expansion granting three new
indications, including routine prevention of angioedema attacks in
children (ages 6 years and above) with severe and recurrent attacks
of HAE.[7]
About the CINRYZE Pediatric
Study
Study 0624-301 was a Phase 3 multicenter, single-blind study that
enrolled 12 patients aged 7 to 11 with HAE who were required to
have an average of greater than or equal to1.0 angioedema attacks
per month that were moderate, severe, or required acute treatment
during the 12-week baseline observation period. Patients received
500 U and 1000 U of CINRYZE every 3-4 days for 12 weeks. The
primary efficacy endpoint was the monthly-normalized number of
attacks.
Overall the safety and tolerability of CINRYZE has
been shown to be similar in clinical studies of pediatric,
adolescent and adult patients with HAE.
About CINRYZE
CINRYZE is currently approved for routine prophylaxis against
angioedema attacks in children aged 6 years and older, adolescent
and adult patients with HAE. The active substance in CINRYZE is
C1-Esterase Inhibitor (C1-INH), which raises plasma levels of
C1-INH in patients with HAE, who are prone to swelling due to an
underlying deficiency in C1-INH. Treatment with C1-INH addresses
the underlying cause of HAE by replacing the deficient protein and
helps regulate the production of bradykinin released during an
attack. CINRYZE was the first C1-INH proven to help prevent
swelling attacks in those living with HAE.1
In Europe, CINRYZE is the first and only
C1-Esterase Inhibitor (C1-INH) therapy approved for routine
prevention in children, adolescents, and adults with HAE. CINRYZE
is also approved for acute treatment and pre-procedure prevention
of angioedema attacks.
IMPORTANT SAFETY INFORMATION
(U.S. ONLY)
Contraindications: CINRYZE is contraindicated in
patients who have manifested life-threatening immediate
hypersensitivity reactions, including anaphylaxis, to the
product.
Hypersensitivity
Reactions: Severe hypersensitivity reactions may occur during
or after administration of CINRYZE. Consider treatment methods
carefully, because hypersensitivity reactions may have symptoms
similar to HAE attacks. In case of hypersensitivity, discontinue
CINRYZE infusion and institute appropriate treatment. Have
epinephrine immediately available for treatment of an acute severe
hypersensitivity reaction.
Thromboembolic
Events: Serious arterial and venous thromboembolic (TE) events
have been reported at the recommended dose of C1 Esterase Inhibitor
(Human) products, including CINRYZE, following administration in
patients with HAE. Risk factors may include presence of an
indwelling venous catheter/access device, prior history of
thrombosis, underlying atherosclerosis, use of oral contraceptives,
certain androgens, morbid obesity, and immobility. Benefits of
CINRYZE for routine prophylaxis of HAE attacks should be weighed
against the risks of TE events in patients with underlying risk
factors. Monitor patients with known risk factors for TE events
during and after CINRYZE administration.
Transmissible
Infectious Agents: Because CINRYZE is made from human blood, it
may carry a risk of transmitting infectious agents, e.g. viruses,
and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. ALL
infections thought by a physician possibly to have been transmitted
by CINRYZE should be reported to Shire Medical Information
at
1-800-828-2088.
Adverse Reactions: The
only serious adverse reaction observed in clinical studies of
CINRYZE was cerebrovascular accident. The most common adverse
reactions (greater than or equal to 5%) observed were headache,
nausea, rash, vomiting, and fever. Post marketing adverse reactions
include local infusion site reactions and hypersensitivity.
Post marketing thromboembolic events have been reported,
including catheter-related and deep venous thromboses, transient
ischemic attack, and stroke.
Please click here for Full Prescribing Information
To report
SUSPECTED ADVERSE REACTIONS, contact Shire Medical Information at
1-800-828-2088 or FDA at 1-800-FDA-1088 or
www.fda.gov/medwatch.
Shire's Commitment to Hereditary
Angioedema
HAE is a rare, genetic disorder
estimated to affect about 1 in 10,000 to 1 in 50,000 people
worldwide.2,3 The
condition results in recurring attacks of edema (swelling) in
various parts of the body, including the abdomen, face, feet,
genitals, hands and throat that can be debilitating and painful.
Attacks that obstruct the airways (asphyxiation) are potentially
life-threatening.2,4,5 Shire is
a dedicated, long-term partner to the HAE community with a decade
of experience supporting patients. We are committed to serial
innovation in HAE and our portfolio of products includes a number
of therapy options to help meet the individual needs of those
living with the disease. Beyond our focus on developing novel
treatments, we provide specialized services and support offerings
tailored to the HAE community. Learn more at
shire.com.
For further information please
contact:
Investor Relations |
|
|
Christoph Brackmann |
christoph.brackmann@shire.com |
+41 795 432 359 |
Sun Kim |
sun.kim@shire.com |
+1 617 588 8175 |
Scott Burrows |
scott.burrows@shire.com |
+41 41 288 4195 |
|
|
|
Media |
|
|
Katie Joyce |
kjoyce@shire.com |
+1 781 482 2779 |
|
|
|
NOTES TO EDITORS
About Shire
Shire is the global biotechnology leader serving
patients with rare diseases and specialized conditions. We seek to
push boundaries through discovering and delivering new
possibilities for patient communities who often have few or no
other champions. Relentlessly on the edge of what's next, we are
serial innovators with a diverse pipeline offering fresh thinking
and new hope. Serving patients and partnering with healthcare
communities in over 100 countries, we strive to be part of the
entire patient journey to enable earlier diagnosis, raise standards
of care, accelerate access to treatment, and support patients. Our
Rare Disease and Neuroscience divisions support our diverse
portfolio of therapeutic areas, including Immunology, Hematology,
Genetic Diseases, Internal Medicine, Ophthalmics, Oncology, and
neuropsychiatry disorders.
Championing patients is our call to action - it brings the
opportunity - and responsibility - to change people's lives.
www.shire.com
Stephen Williams. Deputy Company Secretary, is responsible for
arranging the release of this announcement.
Insider
Information
This announcement contains inside
information.
Forward-Looking
Statements
Statements included herein that are not historical facts, including
without limitation statements concerning future strategy, plans,
objectives, expectations and intentions, projected revenues, 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;
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Shire depends on third parties to supply certain
inputs and services critical to its operations including certain
inputs, services and ingredients critical to its manufacturing
processes. 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;
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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, among
other things, significant delays, an increase in operating costs,
lost product sales, an interruption of research activities or the
delay of new product launches;
-
the nature of producing plasma-based therapies
may prevent Shire from timely responding to market forces and
effectively managing its production capacity;
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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;
-
failure to comply with laws and regulations
governing the sales and marketing of its products could materially
impact Shire's revenues and profitability;
-
Shire's products and product candidates face
substantial competition in the product markets in which it
operates, including competition from generics;
-
Shire's patented products are subject to
significant 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 Shire's
revenues, financial condition or results of operations;
-
Shire may fail to obtain, maintain, enforce or
defend the intellectual property rights required to conduct its
business;
-
Shire faces intense competition for highly
qualified personnel from other companies and organizations;
-
failure to successfully execute or attain
strategic objectives from Shire's acquisitions and growth strategy
may adversely affect the 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,
could have negative consequences for Shire's business and increase
the risk of non-payment by Shire's customers;
-
changes in foreign currency exchange rates and
interest rates could have a material adverse effect on Shire's
operating results and liquidity;
-
Shire is subject to evolving and complex tax
laws, which may result in additional liabilities that may adversely
affect the Shire's financial condition or results of
operations;
-
if a marketed product fails to work effectively
or causes adverse side effects, this could result in damage to
Shire's reputation, the withdrawal of the product and legal action
against Shire;
-
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 faces risks relating to the expected exit
of the United Kingdom from the European Union;
-
Shire incurred substantial additional
indebtedness to finance the Baxalta acquisition, which has
increased its borrowing costs and may decrease its business
flexibility;
-
Shire's ongoing strategic review of its
Neuroscience franchise may distract management and employees and
may not lead to improved operating performance or financial
results; there can be no guarantee that, once completed, Shire's
strategic review will result in any additional strategic changes
beyond those that have already been announced;
-
the potential uncertainty resulting from the
announcement by Takeda Pharmaceutical Company Limited on 8 May 2018
of a recommended offer for Shire under the UK Takeover Code;
and
a further list and description of risks,
uncertainties and other matters can be found in Shire's most recent
Annual Report on Form 10-K and in Shire's subsequent Quarterly
Reports on Form 10-Q, in each case including those risks outlined
in "ITEM1A: Risk Factors", and in Shire's subsequent reports on
Form 8-K and other Securities and Exchange Commission filings, all
of which are available on Shire's website.
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.
[1] CINRYZE (C1
Esterase Inhibitor [Human]) Prescribing Information; 2018.
[2] Cicardi M,
Bork K, Caballero T, et al, on behalf of HAWK (Hereditary
Angioedema International Working Group). Evidence-based
recommendations for the therapeutic management of angioedema owing
to hereditary C1 inhibitor deficiency: consensus report of an
International Working Group. Allergy. 2012; 67(2):147-157.
[3] Longhurst
HJ, Bork K. Hereditary angioedema: causes, manifestations, and
treatment. Br J Hosp Med. 2006;67(12):654-657.
[4] Zuraw BL.
Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.
[5] Banerji A.
The burden of illness in patients with hereditary angioedema. Ann
Allergy Asthma Immunol. 2013;111(5):329-336.
[6] Farkas H,
et al. International consensus on the diagnosis and management of
paediatric patients with hereditary angioedema with C1 inhibitor
deficiency. Allergy. 2017; 72(2):300-13.
[7] CINRYZE (C1
Esterase Inhibitor [Human]) EPAR Prescribing Information; 2017.
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The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: Shire plc via Globenewswire
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