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

Shire
Posted on: 26 Jun 17

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. 

Editor's Details

Mike Wood
PharmiWeb.com
www.pharmiweb.com
editor@pharmiweb.com

Last updated on: 26/06/2017

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