CHMP recommends EU marketing authorization for ADYNOVI® [Antihemophilic Factor (Recombinant), PEGylated] for adults and adolescents with Hemophilia A
(Thomson Reuters ONE) -
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 episodes(1)
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:
* Are allergic to mice or hamster protein
* Are allergic to any ingredients in ADYNOVATE or ADVATE [Antihemophilic
Factor (Recombinant)]
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(at)shire.com +44 203 549 0874
Ian Karp ikarp(at)shire.com +1 781 482 9018
Media
Gwen Fisher gfisher(at)shire.com +1 781 482 9649
Molly Poarch molly.poarch(at)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
1. 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.
2. 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.
3. 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.
4. 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.
5. 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.
6. World Federation of Hemophilia. What is hemophilia? Available at:
http://www.wfh.org/en/page.aspx?pid=646 Last accessed October 2017.
7. 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.
8. National Hemophilia Foundation. Fast facts. 2017. Available at:
https://www.hemophilia.org/About-Us/Fast-Facts Last accessed October 2017.
9. 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 of Nasdaq Corporate Solutions clients.
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
Datum: 13.11.2017 - 07:00 Uhr
Sprache: Deutsch
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