ZUG, Switzerland, June 22, 2017 /PRNewswire/ --
VEYVONDI is the first and only
recombinant von Willebrand factor treatment for adults with von
Willebrand disease, the most common inherited bleeding
disorder[1],[2],[3]
Shire plc (LSE: SHP, NASDAQ: SHPG), the leading biotechnology
company focused on serving individuals with rare diseases, today
announced that the European Medicines Agency (EMA) validated
the Marketing Authorization Application (MAA) for VEYVONDI to
prevent and treat bleeding episodes and peri-operative bleeding in
adults (age 18 and older) diagnosed with von Willebrand Disease
(VWD), the most common inherited bleeding disorder. Currently
available in the U.S. as VONVENDI® [von Willebrand
factor (Recombinant)], VEYVONDI is the first and only recombinant
von Willebrand factor (rVWF) treatment for adults living with
VWD.[1],[2],[3]
"With its clinical profile and physiology, VEYVONDI is a
first-of-its-kind treatment for VWD," said Alice Dietrich, MD, Head of Global Medical
Affairs, Shire. "We're deeply committed to advancing standards of
care and outcomes for VWD patients worldwide, and today's
submission marks an important milestone in our efforts to address
needs of patients living with VWD and make VEYVONDI available to
more patients who could benefit from its use."
Patients with VWD have a deficiency or dysfunction of VWF, a
blood protein required for proper
clotting.[4] Because of this, the
blood does not clot properly, which may result in heavy menstrual
periods, easy bruising, or frequent nose
bleeds.[4] VEYVONDI is an
innovative VWF replacement therapy produced and formulated without
the addition of any exogenous raw materials of human or animal
origin, resulting in a product that contains only trace amounts of
FVIII.[2] This provides physicians
with the flexibility to manage VWF
levels.[3]
VEYVONDI was studied in patients, 18 to 64 years of age, in a
multi-center, open label, non-randomized study assessing safety,
efficacy and pharmacokinetics, with and without
rFVIII.[2],[3]
It was also studied in patients, 18 years and older, in a
prospective, uncontrolled, international, multi-center, open label,
non-randomized study assessing control of hemostasis before, during
or after surgical procedures, with or without
rFVIII.[5] The most common adverse
reaction observed was generalized pruritus
(itching).[3],[6]
The EMA filing is based on data from these two Phase 3 clinical
trials shared publicly in December
2015 and December 2016,
respectively.
About von Willebrand disease (VWD)
VWD is the most common inherited bleeding disorder worldwide,
affecting up to 1 percent of the world's
population.[7] VWD is caused by a
deficiency or dysfunction of VWF, resulting in impaired blood
clotting, and it affects women and men
equally.[4],[8]
The disease can manifest through various bleeding events, including
heavy menstrual periods, easy bruising or frequent nose
bleeds.[4] Bleeding caused by VWD
is unpredictable and varies greatly among patients with the
disease.[8] Many people with VWD
may not know that they have it because their symptoms are mild or
change over time.[9] Research
shows that as many as 9 out of 10 people with VWD have not been
diagnosed.[9]
About VONVENDI in the
United States
VONVENDI was approved by the U.S. Food and Drug Administration
(FDA) in December 2015 and is
indicated for on-demand treatment and control of bleeding episodes
in adults (18 years and older) with
VWD.[1] VONVENDI is the first and
only rVWF treatment developed for people living with
VWD.[1] Shire is also seeking
prophylaxis and pediatric indications for VONVENDI, with trials
anticipated to conclude in 2019 and 2020, respectively. For more
information on VONVENDI, please visit vonvendi.com.
VONVENDI [von Willebrand factor (Recombinant)] Important
Information
Indication
VONVENDI [von Willebrand factor (Recombinant)] is a recombinant von
Willebrand factor indicated for on-demand treatment and control of
bleeding episodes in adults (age 18 and older) diagnosed with von
Willebrand disease.
DETAILED IMPORTANT RISK INFORMATION
CONTRAINDICATIONS
VONVENDI is contraindicated in patients who have had
life-threatening hypersensitivity reactions to VONVENDI or
constituents of the product (tri-sodium citrate-dihydrate, glycine,
mannitol, trehalose-dihydrate, polysorbate 80, and hamster or mouse
proteins).
WARNINGS AND PRECAUTIONS
Embolism and Thrombosis
Thromboembolic reactions, including disseminated intravascular
coagulation (DIC), venous thrombosis, pulmonary embolism,
myocardial infarction, and stroke, can occur, particularly in
patients with known risk factors for thrombosis. Monitor for early
signs and symptoms of thrombosis such as pain, swelling,
discoloration, dyspnea, cough, hemoptysis, and syncope.
In patients requiring frequent doses of VONVENDI with
recombinant factor VIII, monitor plasma levels for FVIII:C activity
because an excessive rise in factor VIII levels can increase the
risk of thromboembolic complications.
Hypersensitivity Reactions
Hypersensitivity reactions, including anaphylaxis, may occur.
Symptoms can include anaphylactic shock, generalized urticaria,
angioedema, chest tightness, hypotension, shock, lethargy, nausea,
vomiting, paresthesia, pruritus, restlessness, wheezing and/or
acute respiratory distress. If signs and symptoms of severe
allergic reactions occur, immediately discontinue administration of
VONVENDI and provide appropriate supportive care.
Neutralizing Antibodies
Neutralizing antibodies (inhibitors) to von Willebrand factor
and/or factor VIII can occur. If the expected plasma levels of VWF
activity (VWF:RCo) are not attained, perform an appropriate assay
to determine if anti-VWF or anti-FVIII inhibitors are present.
Consider other therapeutic options and direct the patient to a
physician with experience in the care of either von Willebrand
disease or hemophilia A.
In patients with high levels of inhibitors to VWF or factor
VIII, VONVENDI therapy may not be effective and infusion of this
protein may lead to severe hypersensitivity reactions. Since
inhibitor antibodies can occur concomitantly with anaphylactic
reactions, evaluate patients experiencing an anaphylactic reaction
for the presence of inhibitors.
ADVERSE REACTIONS
The most common adverse reaction observed in ≥2% of subjects in
clinical trials (n=66) was generalized pruritus.
For Full Prescribing Information,
visit http://www.shirecontent.com/PI/PDFs/VONVENDI_USA_ENG.pdf
SHIRE and the Shire Logo are registered trademarks of Shire
Pharmaceutical Holdings Ireland Limited or its affiliates. VEYVONDI
and VONVENDI are trademarks or registered trademarks 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
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- 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 acquisition of 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
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- a slowdown of global economic growth, or economic instability
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disruptions, the loss of sensitive or confidential information,
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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; 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 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
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information, future events or otherwise.
References
- "FDA approved first recombinant von Willebrand factor to treat
bleeding episodes." FDA website.
https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm476065.
Accessed June 21, 2017.
- VONVENDI Prescribing Information.
- Gill JC, Castaman G, Windyga J, et al. Hemostatic efficacy,
safety, and pharmacokinetics of a recombinant von Willebrand factor
in severe von Willebrand disease. Blood.
2015;126(17):2038-2046.
- Nichols WL, Hultin MB, James AH, et al. von Willebrand disease
(VWD): evidence-based diagnosis and management guidelines, the
National Heart, Lung, and Blood Institute (NHLBI) Expert Panel
report (USA). Haemophilia.
2008;14(2):171-232.
- Baxalta. Recombinant von Willebrand factor in subjects with
severe von Willebrand disease undergoing surgery. Available from:
https://clinicaltrials.gov/ct2/show/NCT02283268. Accessed
June 16, 2017.
- Shire Data on File.
- James PD, Goodeve AC. von Willebrand Disease. Genet Med.
2011;13(5):365-76.
- Leebeek FW, Eikenboom JC. Von Willebrand's Disease. N Engl J
Med. 2016;375(21):2067-2080.
- What is von Willebrand disease (VWD)? World Federation of
Hemophilia website. https://www.wfh.org/en/page.aspx?pid=673.
Accessed May 3, 2017.
For further information, please contact:
Investor Relations
Ian Karp
ikarp@shire.com
+1-781-482-9018
Robert Coates
rcoates@shire.com
+44-1256-894874
Media
Molly Poarch
Molly.poarch@shire.com
+1-312-965-3414