YASTEST
CHMP recommends EU marketing
authorization for ADYNOVI®
[Antihemophilic Factor (Recombinant), PEGylated] for adults and
adolescents with Hemophilia A
If approved,
ADYNOVI, built on ADVATE®
[Antihemophilic Factor (Recombinant)], would offer
twice-weekly prophylactic dosing as well as on-demand control of
bleeding episodes1
Zug, Switzerland - November 13,
2017 - Shire plc (LSE: SHP, NASDAQ: SHPG), the world's leading
biotechnology company in rare diseases, announced today that the
Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency (EMA) has issued a positive opinion
recommending the granting of marketing authorization for ADYNOVI
[Antihemophilic Factor (Recombinant), PEGylated], an extended
half-life recombinant Factor VIII (rFVIII) treatment, for adults
and adolescents ages 12 and older living with hemophilia A for
on-demand and prophylactic use.1
"This positive opinion marks an important step in
providing adults and adolescents with hemophilia A in Europe with
the potential for a new treatment option with a twice-weekly
prophylaxis dosing schedule, so patients can manage their
hemophilia A care with their physicians in the way that works best
for them" said Howard B. Mayer, M.D., SVP and ad-interim Head,
Global Research and Development, Shire.
The CHMP submission was based on outcomes from
three Phase 3 clinical trials of patients with hemophilia A. These
include a prospective, global, multi-centre, open label,
non-randomized study of patients 12 to 65 years of age; a
prospective, uncontrolled, open label, multi-centre study of
patients 12 years of age and younger; and a study of perioperative
control of hemostasis with interim study results from 15 patients
with severe hemophilia A undergoing surgical procedures.2,3,4
Hemophilia A, a designated orphan disease by the
European Commission (EC), is a rare bleeding disorder that causes
longer-than-normal bleeding due to lack of clotting factor VIII
(FVIII) in the blood.5,6
Today, hemophilia A affects more than 150,000 people across the
globe.7 It primarily
affects males, with an incidence of one in 5,000 male
births.8 Of the
worldwide hemophilia patient population, an estimated 75 percent
lack adequate treatment or access to treatment
altogether.8
The CHMP's Positive Opinion will be reviewed by
the European Commission, which has the authority to grant marketing
authorization in the EU.
About Hemophilia A
Hemophilia A, the most common type of hemophilia, is a rare
bleeding disorder that causes longer-than-normal bleeding due to
lack of clotting factor VIII (FVIII) in the blood.6
The severity of hemophilia A is determined by the amount of factor
in the blood, with more severity associated with lower amounts of
factor.9 More than
half of patients with hemophilia A have the severe form of the
condition.9
About ADYNOVI
ADYNOVI [Antihemophilic Factor (Recombinant), PEGylated] was first
approved as ADYNOVATE® by the Food
and Drug Administration (FDA) in the U.S. followed by approval in
Japan, Canada, and Colombia, and is approved as ADYNOVI in
Switzerland.
For more information on ADYNOVI, please visit the
EMA website.
U.S. IMPORTANT INFORMATION FOR
ADYNOVATE
What is ADYNOVATE?
-
ADYNOVATE is an injectable medicine that is used
to help treat and control bleeding in children and adults with
hemophilia A (congenital Factor VIII deficiency).
-
Your healthcare provider may give you ADYNOVATE
when you have surgery.
-
ADYNOVATE can reduce the number of bleeding
episodes when used regularly (prophylaxis).
ADYNOVATE is not used to treat von Willebrand
disease.
DETAILED IMPORTANT RISK
INFORMATION
You should not use ADYNOVATE if you:
Tell your healthcare provider if you are pregnant
or breastfeeding because ADYNOVATE may not be right for you.
You should tell your healthcare provider if
you:
-
Have or have had any medical problems.
-
Take any medicines, including prescription and
non-prescription medicines, such as over-the-counter medicines,
supplements or herbal remedies.
-
Have any allergies, including allergies to mice
or hamsters.
-
Have been told that you have inhibitors to
factor VIII (because ADYNOVATE may not work for you).
Your body may form inhibitors to Factor VIII. An
inhibitor is part of the body's normal defense system. If you form
inhibitors, it may stop ADYNOVATE from working properly. Consult
with your healthcare provider to make sure you are carefully
monitored with blood tests for the development of inhibitors to
Factor VIII.
You can have an allergic reaction to ADYNOVATE.
Call your healthcare provider right away and stop treatment if you
get a rash or hives, itching, tightness of the throat, chest pain
or tightness, difficulty breathing, lightheadedness, dizziness,
nausea or fainting.
The common side effects of ADYNOVATE are headache
and nausea. Tell your healthcare provider about any side effects
that bother you or do not go away.
For additional safety information, click here for
Prescribing Information and discuss with your doctor.
You are encouraged to report
negative side effects of prescription drugs to the FDA. Visit
www.fda.gov/medwatch, or call 1-800-FDA-1088.
About ADVATE
ADVATE is currently approved in 69 countries
worldwide, including the United States and 28 countries in the
European Union.
EU IMPORTANT INFORMATION FOR
ADVATE
Please consult the Advate Summary Product
Characteristics (SPC) before prescribing, particularly in relation
to dosing and treatment monitoring.
Contraindications include hypersensitivity to the
active substance or to any of the excipients listed in SmPC section
6.1 or to mouse or hamster proteins.
The ADRs that occurred in the highest frequency
were development of neutralising antibodies to factor VIII
(inhibitors), headache and pyrexia.
Hypersensitivity or allergic reactions have been
observed rarely and may in some cases progress to severe
anaphylaxis (including shock).
U.S. IMPORTANT INFORMATION FOR
ADVATE
What is ADVATE?
-
ADVATE is a medicine used to replace clotting
factor (factor VIII or antihemophilic factor) that is missing in
people with hemophilia A (also called "classic" hemophilia).
-
ADVATE is used to prevent and control bleeding
in adults and children (0-16 years) with hemophilia A. Your
healthcare provider may give you ADVATE when you have
surgery.
-
ADVATE can reduce the number of bleeding
episodes in adults and children (0-16 years) when used regularly
(prophylaxis).
ADVATE is not used to treat von Willebrand
disease.
For additional safety information, click here for
Prescribing Information and discuss with your doctor.
You are encouraged to report
negative side effects of prescription drugs to the FDA. Visit
www.fda.gov/medwatch, or call 1-800-FDA-1088.
FOR FURTHER
INFORMATION, PLEASE CONTACT:
Investor Relations |
|
|
Robert Coates |
rcoates@shire.com |
+44 203 549 0874 |
Ian Karp |
ikarp@shire.com |
+1 781 482 9018 |
|
|
|
Media |
|
|
Gwen Fisher |
gfisher@shire.com |
+1 781 482 9649 |
Molly Poarch |
molly.poarch@shire.com |
+1 312 965 3413 |
NOTES TO EDITORS
About Shire
Shire is the global leader in serving patients
with rare diseases. We strive to develop best-in-class therapies
across a core of rare disease areas including hematology,
immunology, genetic diseases, neuroscience, and internal medicine
with growing therapeutic areas in ophthalmics and oncology. Our
diversified capabilities enable us to reach patients in more than
100 countries who are struggling to live their lives to the
fullest.
We feel a strong sense of urgency to address unmet
medical needs and work tirelessly to improve people's lives with
medicines that have a meaningful impact on patients and all who
support them on their journey.
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, 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;
-
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 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,
including expected operating efficiencies, cost savings, revenue
enhancements, synergies or other benefits at the time anticipated
or at all with respect to Shire's acquisitions, including NPS
Pharmaceuticals Inc., Dyax Corp. or Baxalta Incorporated 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 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 has
increased its borrowing costs may decrease its business
flexibility; 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 "ITEM 1A: 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.
###
References
-
European Medicines Agency. CHMP Summary of
Opinion: Adynovi. Available at:
http://www.ema.europa.eu/docs/en_GB/document_library/Summary_of_opinion_-_Initial_authorisation/human/004195/WC500238103.pdf.
Last accessed November 2017.
-
Konkle BA, Stasyshyn O, Chowdary P, et al. Pegylated, full-length, recombinant factor VIII
for prophylactic and on-demand treatment of severe hemophilia A.
Blood. 2015;126:1078-85.
-
Mullins ES, Stasyshyn O, Alvarez-Román MT,
et al. Extended half-life pegylated,
full-length recombinant factor VIII for prophylaxis in children
with severe haemophilia A. Haemophilia.
2017;23:238-246.
-
Brand B, Gruppo R, Wynn TT, et al. Efficacy and
safety of pegylated full-length recombinant factor VIII with
extended half-life for perioperative haemostasis in haemophilia A
patients. Haemophilia. 2016;22:e251-8.
-
Orphanet. Rare disease registries in Europe.
Orphanet Report Series: Rare Disease Collection, May 2017.
Available at:
http://www.orpha.net/orphacom/cahiers/docs/GB/Registries.pdf Last
accessed October 2017.
-
World Federation of Hemophilia. What is
hemophilia? Available at: http://www.wfh.org/en/page.aspx?pid=646
Last accessed October 2017.
-
World Federation of Hemophilia. Report on the
Annual Global Survey 2015. October 2016. Available at:
https://www1.wfh.org/publication/files/pdf-1669.pdf Last accessed
October 2017.
-
National Hemophilia Foundation. Fast facts.
2017. Available at: https://www.hemophilia.org/About-Us/Fast-Facts
Last accessed October 2017.
-
National Hemophilia Foundation. Hemophilia A.
Available at:
https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A
Last accessed October 2017.
This
announcement is distributed by Nasdaq Corporate Solutions on behalf
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The issuer of this announcement warrants that they are solely
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information contained therein.
Source: Shire plc via Globenewswire
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