Shire Pharmaceuticals Group Plc

Shire Announces EMA Validation of Veyvondi[TM] [Von Willebrand Factor (Recombinant)] Marketing Authorization Application for Treatment of Von Willebrand Disease

Zug, Switzerland (ots/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 [von Willebrand factor (Recombinant)] Important Information


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.



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).

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.


The most common adverse reaction observed in >=2% of subjects in clinical trials (n=66) was generalized pruritus.

For Full Prescribing Information, visit

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.


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.

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 
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- Shire conducts its own manufacturing operations for certain of its 
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- certain of Shire's therapies involve lengthy and complex processes,
  which may prevent Shire from timely responding to market forces and
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- inability to successfully compete for highly qualified personnel 
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  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;
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  its product portfolio through external collaborations, which, if 
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- a slowdown of global economic growth, or economic instability of 
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  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 revise forward-looking statements, whether as a result of new information, future events or otherwise.


1. "FDA approved first recombinant von Willebrand factor to treat 
   bleeding episodes." FDA website.
   wsroom/pressannouncements/ucm476065. Accessed June 21, 2017.
2. VONVENDI Prescribing Information.
3. 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. 
4. 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.
5. Baxalta. Recombinant von Willebrand factor in subjects with severe
   von Willebrand disease undergoing surgery. Available from: Accessed June 16,
6. Shire Data on File.
7. James PD, Goodeve AC. von Willebrand Disease. Genet Med. 
8. Leebeek FW, Eikenboom JC. Von Willebrand's Disease. N Engl J Med. 
9. What is von Willebrand disease (VWD)? World Federation of 
   Hemophilia website. 
   Accessed May 3, 2017. 


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Investor Relations
Ian Karp

Robert Coates

Molly Poarch

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