VONVENDI [von Willebrand factor (Recombinant)] is a significant advancement for the treatment of adults with von Willebrand disease (VWD)
Lexington, Mass. – Aug, 9, 2016 – Shire plc (LSE: SHP, NASDAQ: SHPG), continuing on Baxalta’s long-standing commitment to the bleeding disorder community, today announced the U.S. launch of VONVENDI [von Willebrand factor (Recombinant)], the only recombinant treatment for adults living with von Willebrand disease (VWD).
As the first and only recombinant von Willebrand factor (VWF) treatment, VONVENDI replaces the VWF the body is missing in VWD. The treatment is also the first in the United States that has the option to dose recombinant von Willebrand factor (rVWF) independent of recombinant Factor VIII (rFVIII), based on patient need. This attribute allows for tailored treatment for patients who may not require additional FVIII.
VWD is a hereditary bleeding disorder that is caused by deficiency or dysfunction of VWF, a plasma protein that mediates the initial adhesion of platelets at sites of vascular injury and also carries and protects factor VIII from premature proteolysis. Because of this, the blood does not clot properly, resulting in heavy menstrual periods, easy bruising, or frequent nose bleeds. It is estimated that one in 100 people, approximately 3 million people in the United States, suffer from VWD.
“As the first and only recombinant treatment option, VONVENDI represents an important step forward in the treatment of VWD,” said Ralph Gruppo, M.D., Director, Comprehensive Hemophilia and Thrombosis Center at the Cincinnati Children’s Hospital. “By being able to replace with only VWF recombinant proteins that function similarly to VWF that occurs naturally in the body, patients and their physicians have an effective new treatment option for this challenging and chronic disease.”
With the availability of VONVENDI, Shire continues to grow its industry-leading hematology portfolio, invest in treatment options and support for VWD patients around the world, and aspires to help unite the VWD community to elevate awareness and education. Regulatory submission in Europe is expected in 2017 and in other markets globally. Additionally, VONVENDI is being studied for use in the surgical setting.
“VONVENDI marks a new era in treating von Willebrand disease to help give people with VWD control over their bleeds,” said Shannon Resetich, Head of Global Hematology, Shire. “We look forward to expanding availability to patients who can benefit from this treatment all over the world.”
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 (age 18 and older) with VWD. The FDA approval was based on positive results from a Phase 3 multicenter, open-label clinical trial that assessed the safety, efficacy and pharmacokinetics of VONVENDI with and without recombinant FVIII. In the pivotal study, all participants (100 percent) reported successful treatment of bleeding episodes, with 96.9 percent of treated bleeds (N=192 bleeds in 22 patients) achieving an “excellent” efficacy rating and 3.1 percent achieving a “good” efficacy rating. The most common adverse reaction observed in greater than two percent of patients in clinical trials was generalized pruritus. For more information on VONVENDI, please visit Vonvendi.com.
Important Information for VONVENDI [von Willebrand factor (Recombinant)]
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 for VONVENDI
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.
Please see VONVENDI full Prescribing Information at http://www.baxalta.com/assets/documents/VONVENDI_PI.pdf
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.
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