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Shire Granted EU Marketing Authorization for Veyvondi® for Adults With Von Willebrand Disease

SHIRE GRANTED EU MARKETING AUTHORIZATION FOR VEYVONDI® [VONICOG ALFA, RECOMBINANT VON WILLEBRAND FACTOR] FOR ADULTS WITH VON WILLEBRAND DISEASE

  • Marketing Authorization will enable patient access to VEYVONDI® throughout Europe
  • VEYVONDI [vonicog alfa, recombinant von Willebrand factor] is the first and only recombinant von Willebrand Factor (rVWF) to treat hemorrhage and treat/prevent surgical bleeding in adults (age 18 and older) with von Willebrand disease (VWD)1

/EIN News/ -- Zug, Switzerland – September 12, 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG) the global biotech leader in rare diseases, announced today that the European Commission (EC) has granted Marketing Authorization for VEYVONDI [vonicog alfa, recombinant von Willebrand factor] (rVWF), for the treatment of bleeding events and treatment/prevention of surgical bleeding in adults (age 18 and older) with von Willebrand disease (VWD) when desmopressin (DDAVP) treatment alone is ineffective or not indicated. VEYVONDI should not be used in the treatment of hemophilia A.1 VEYVONDI is the first and only recombinant von Willebrand Factor (rVWF) treatment in the EU for von Willebrand disease (VWD) that specifically addresses the primary deficiency or dysfunction of von Willebrand Factor (VWF) while also allowing the body to restore and maintain adequate Factor VIII (FVIII) plasma levels.1

“The approval in Europe for VEYVONDI marks a key milestone in our efforts to tackle unmet medical needs for those living with von Willebrand disease,” said Andreas Busch, Head of Research and Development and Chief Scientific Officer, Shire. “We are excited to take the next steps in ensuring that VEYVONDI is widely available across Europe to address the individual needs of those affected by the condition and in need of factor replacement."

The Marketing Authorization is based on outcomes from three clinical trials of a total 80 patients with VWD exposed to VEYVONDI. These include a Phase 1 multicenter, controlled, randomized, single-blind, dose-escalation study of the safety, tolerability and pharmacokinectics (PK) of rVWF:rFVIII in subjects 18 to 60 years of age with severe VWD; a Phase 3 multicenter, open-label study to assess the PK, safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding episodes in adult subjects with severe VWD; and a Phase 3, prospective, open-label, uncontrolled, non-randomized, international multicenter study to assess the hemostatic efficacy and safety of rVWF with or without rFVIII in 15 adult subjects with severe VWD undergoing major, minor, or oral elective surgical procedures.2

VWD is the most common inherited bleeding disorder, affecting up to 1 percent of the global population or approximately 100,000 people in the EU.3,4 VWD is caused by a deficiency or dysfunction of VWF, one of several types of proteins in the blood that are needed to facilitate proper blood clotting.5 Only a minor proportion of affected individuals have the severe form of the disease and are in need of VWF replacement.6 Symptoms range from nosebleeds to bleeding from the gums and easy bruising. Bleeding from the stomach and intestines can occur but is less common.7

With this approval, Shire is now authorized to market VEYVONDI in the 28 Member States of the European Union (EU), as well as in Iceland, Lichtenstein and Norway.

About von Willebrand disease (VWD)
VWD is the most common inherited bleeding disorder, affecting up to 1 percent of the global population.3 VWD is caused by a deficiency or dysfunction of VWF, one of several types of proteins in the blood that are needed to facilitate proper blood clotting.5 Due to this deficiency or dysfunction in VWF, blood is not able to clot effectively in people with VWD.6 The majority of cases (70-80%) are due to partial quantitative VWF deficiency (Type 1 VWD).8 The most severe form of VWD (severe Type 3 VWD) is the least common, with incidence varying by ethnicity from 0.1 to 5.3 cases per million.9 The mutation may occur spontaneously with no previous family history.7 Bleeding caused by VWD is unpredictable and its severity varies greatly among patients with this disease.10

About VEYVONDI
VEYVONDI is indicated in adults (age 18 and older) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or not indicated for the treatment of hemorrhage and treatment/prevention of surgical bleeding. VEYVONDI should not be used in the treatment of hemophilia A.1

For full EU Summary of Product Characteristics, including approved indication(s) and important safety information about marketed products, please visit: http://ec.europa.eu/health/documents/community-register/2018/20180831141871/anx_141871_en.pdf1

In the US the product has been approved for more than two years under the trade name VONVENDI® [von Willebrand factor (Recombinant)] and is indicated for use in adults (age 18 and older) diagnosed with von Willebrand disease for on-demand treatment and control of bleeding episodes or perioperative management of bleeding.

VEYVONDI EU IMPORTANT SAFETY INFORMATION1

WARNINGS AND PRECAUTIONS
Hypersensitivity Reactions
Hypersensitivity reactions (including anaphylaxis) may occur. Patients and/or their caregivers should be informed of the early signs of hypersensitivity reactions, which may include but are not limited to tachycardia, tightness of the chest, wheezing and/or acute respiratory distress, hypotension, generalized urticaria, pruritus, rhinoconjunctivitis, angioedema, lethargy, nausea, vomiting, paresthesia, restlessness, and may progress to anaphylactic shock. In case of shock, standard medical treatment for shock should be implemented.

Patients should be closely monitored and carefully observed for any symptoms throughout the infusion period. If signs and symptoms of severe allergic reactions occur, immediately discontinue administration of VEYVONDI and provide appropriate supportive care. Adequate medical treatment and provisions should be available for immediate use for a potential anaphylactic reaction, especially for patients with a history of allergic reactions.

VEYVONDI contains trace amounts of mouse immunoglobulin G (MuIgG) and hamster proteins (less than or equal to 2 ng/IU VEYVONDI). Patients treated with this product may develop hypersensitivity reactions to these non-human mammalian proteins. VEYVONDI contains trace amounts of recombinant coagulation factor VIII.

Thrombosis and Embolism
There is a risk of occurrence of thrombotic events, particularly in patients with known clinical or laboratory risk factors for thrombosis including low ADAMTS13 levels. Therefore, patients at risk have to be monitored for early signs of thrombosis, and prophylaxis measures against thromboembolism should be instituted according to current recommendations and standard of care.

In patients requiring frequent doses of VEYVONDI in combination with recombinant factor VIII, plasma levels for FVIII:C activity should be monitored to avoid sustained excessive FVIII:C plasma levels, which may increase the risk of thrombotic events. Any FVIII that would be administered along with VEYVONDI should be a pure FVIII product. A combination with a FVIII product containing VWF would pose an additional risk of thrombotic events.

Neutralizing Antibodies (Inhibitors)
Patients with VWD, especially Type 3, may develop neutralising antibodies (inhibitors) to von Willebrand factor. If the expected plasma levels of (VWF:RCo) are not attained, or if bleeding is not controlled with an appropriate dose, an appropriate assay should be performed to determine if a von Willebrand factor inhibitor is present. In patients with high levels of anti-VWF antibodies, von Willebrand factor therapy may not be effective and other therapeutic options should be considered.

Treatment of VWD patients who have high-titer binding antibodies may require a higher dose to overcome the binding antibody effect and such patients could be managed clinically by administration of higher doses of vonicog alfa based on the PK data for each individual patient.

ADVERSE REACTIONS

The most common side effects are dizziness, vertigo, dysgeusia, tremor, tachycardia, deep venous thrombosis, hypertension, hot flush, vomiting, nausea, generalised pruritus, chest discomfort, infusion site paraesthesia, electrocardiogram T wave inversion and increased heart rate.

In clinical trials, one case of clinically asymptomatic deep vein thrombosis (DVT) was reported for a subject in the surgery study who had total hip replacement. In addition, one post-marketing case of DVT has been reported spontaneously for an elderly patient.


For further information please contact:

 

Media
   
Linda Calandra Linda.calandra@shire.com  +1 917 697 7543
Katie Joyce kjoyce@shire.com  +1 781 482 2779
 

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

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 diverse portfolio of therapeutic areas includes Immunology, Hematology, Genetic Diseases, Neuroscience, Internal Medicine, and Ophthalmics.
 
Championing patients is our call to action - it brings the opportunity - and responsibility - to change people’s lives.

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, 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;
  • 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;
  • 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;
  • 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 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 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 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;
  • the potential uncertainty among our employees, customers, suppliers, and other business partners resulting from the announcement by Takeda Pharmaceutical Company Limited on May 8, 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.


REFERENCES

1 European Commission. Veyvondi Summary of Product Characteristics. Available at: http://ec.europa.eu/health/documents/community-register/2018/20180831141871/anx_141871_en.pdf

2 Recombinant von Willebrand Factor; rVWF; vonicog alfa; BAX 111. Clinical Overview. May 2017.

3 World Federation of Hemophilia. What is von Willebrand disease (VWD)? Available at: www.wfh.org/en/page.aspx?pid=673.

4 European Medicines Agency. EU/3/10/814. Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/orphans/2010/12/human_orphan_000865.jsp&mid=WC0b01ac058001d12b.

5 National Hemophilia Foundation. Von Willebrand Disease. Available at: https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Von-Willebrand-Disease.

6 National Organization for Rare Disorders. Von Willebrand Disease. Available at: https://rarediseases.org/rare-diseases/von-willebrand-disease/.

7 Rare Diseases. Von Willebrand Disease. Available at: https://rarediseases.org/rare-diseases/von-willebrand-disease/.

8 Kujovic, J.L. Von Willebrand’s Disease and Menorrhagia: Prevalence, Diagnosis, and Management. Available at: https://onlinelibrary.wiley.com/doi/pdf/10.1002/ajh.20372.

9 Verma, A., Yaish, H. et al. Type 3 Von Willebrand Disease with Alloantibodies and Its Challenging Management During Episode of Bleeding. Available at: http://www.bloodjournal.org/content/116/21/1405.

10 National Hemophilia Foundation. "VWD Summit Highlights." HEMAWARE website. https://hemaware.org/story/vwd-summit-highights.


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