Results will form the basis of a supplemental new drug application to the U.S. Food & Drug Administration, requesting an expanded indication for VONVENDI
Lexington, Mass. – December 2, 2016 – Shire plc (LSE: SHP, NASDAQ: SHPG), the leading biotechnology company focused on serving individuals with rare diseases and other highly specialized conditions, today announced topline results from a Phase 3 clinical trial of VONVENDITM [von Willebrand factor (Recombinant)] to treat bleeds in elective surgical settings for adults with severe von Willebrand disease (VWD), the most common inherited bleeding disorder. VONVENDI is an on-demand recombinant treatment for adults living with VWD and replaces von Willebrand factor (VWF), one of several types of proteins in the blood that are needed to facilitate proper blood clotting missing in patients with VWD.1 The study met its primary endpoint and indicates that VONVENDI effectively controlled bleeding and blood loss during an operation in adults undergoing major, minor and oral elective surgical procedures. The results will form the basis of a supplemental new drug application to the U.S. Food & Drug Administration, of which Shire is requesting for an expanded indication for VONVENDI.2
“These findings suggest that using VONVENDI to treat bleeds during surgery may offer this patient population a new option designed to help meet individual patient needs during surgery,” said Philip J. Vickers, Ph.D., Head of Research and Development, Shire.2 “Shire is committed to advancing research and possible care options for patients with VWD, building on our commitment to improve outcomes for patients with a range of bleeding disorders.”
The prospective, uncontrolled, open-label, nonrandomized, international, multi-center Phase 3 study enrolled 15 patients 18 years and older diagnosed with severe congenital VWD undergoing major and minor elective surgical procedures. The study assessed the safety and hemostatic efficacy of VONVENDI used before, during or after a surgical procedure, with or without, a recombinant Factor VIII (rFVIII) treatment. Overall hemostatic efficacy was rated as “excellent” or “good” for all 15 treated subjects. A total of 12 treatment emergent adverse events (TEAEs) occurred in six patients during or after infusion treatment. Two serious TEAEs occurred in two patients. Ten non-serious TEAEs occurred in six patients. None of the TEAEs that occurred during the study were assessed as severe allergic reaction. In addition, none of the study subjects developed inhibitory antibodies to VONVENDI, rFVIII or binding antibodies to CHO, rFurin or murine IgG.2
The complete results of the study will be presented at an upcoming medical conference. The results will also be used to support the filing for regulatory approvals in Europe in 2017 and in other markets globally. VONVENDI is also being studied for prophylaxis and pediatric indications.
About von Willebrand disease (VWD)
VWD is the most common inherited bleeding disorder, affecting up to 1 percent of the U.S. 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.3 Due to this deficiency in VWF, blood is not able to clot effectively in people with VWD, which results in heavy menstrual periods, easy bruising or frequent nose bleeds.3 Bleeding caused by VWD is unpredictable and varies greatly among patients with this disease.4
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.
DETAILED IMPORTANT RISK INFORMATION
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, 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 (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 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. VONVENDI is a trademark of Baxalta Incorporated, a wholly owned, indirect subsidiary of Shire plc.
1. Shire. “Introducing VONVENDI [von Willebrand factor (Recombinant)].” VONVENDI website. http://www.vonvendi.com/about/why-vonvendi/ Accessed November 16, 2016.
2. Data on File. VONVENDI Phase 3 Surgical Study Report.
3. National Hemophilia Foundation. “Von Willebrand Disease.” National Hemophilia Foundation website. https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Von-Willebrand-Disease Accessed November 16, 2016.
4. National Hemophilia Foundation. “VWD Summit Highlights.” HEMAWARE website. https://hemaware.org/story/vwd-summit-highights Accessed December 1, 2016.
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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.
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