More than 40 presentations and sessions feature broad pipeline and proven factor replacement portfolio, with new innovation on outcomes, safety and efficacy measures
Lexington, Mass. – July 19, 2016 – Shire plc (LSE: SHP, NASDAQ: SHPG), following its combination with Baxalta in June, will affirm its commitment to advancing patient care and scientific innovation at the International Congress of the World Federation of Hemophilia (WFH) in Orlando, Florida, July 24-28, 2016. Data at the Congress this year will showcase the company’s robust hematology portfolio and pipeline.
Shire is committed to introducing new treatments in hemophilia and other bleeding disorders and aims to enhance patients’ experiences with these chronic bleeding disorders. One of our key focus areas is to advance care based on the proven safety and efficacy profile of direct factor replacement and bypassing agents with treatment approaches tailored to patient needs.
“Shire is proud to carry forward our focus on treatment innovation and support for the hematology community,” said Len Valentino, M.D., Head of Global Medical Affairs, Hematology at Shire. “We are continually pursuing new opportunities to build on the proven approach of direct factor replacement and advance the standard of care for patients in our pursuit of a Bleed-free World.”
Advancing Treatment Approaches to Optimize Patient-Centered Care
During WFH 2016, Shire researchers will address the PROPEL study, which compares the safety and efficacy of ADYNOVATE [Antihemophilic Factor [Recombinant], PEGylated] following pharmacokinetic (PK)-guided prophylaxis targeting two different Factor VIII trough levels. The novel design of the study is built upon preliminary data indicating that maintaining higher trough levels may be able to help enhance bleed protection and help more patients reach zero bleeds.
In addition, the company has initiated research on an innovative patient-reported outcome (PRO) measure: Goal Attainment Scaling – Hematology (GOAL-Hēm). The measure is intended to support broader bleeding disorder management by supplementing standard clinical outcome measures with those that are patient-centered, customizable and sensitive to change across patients and patient populations.
Throughout the Congress, Shire will present more than a dozen scientific updates on the company’s broad portfolio of treatments for bleeding disorders, including ADYNOVATE, its latest treatment for hemophilia A patients. ADYNOVATE is an extended half-life factor VIII replacement treatment built on ADVATE [Antihemophilic Factor (Recombinant)] that offers demonstrated results with twice-weekly dosing. The company will also feature VONVENDI [von Willebrand factor (Recombinant)], which launches commercially in the U.S. in the third quarter of 2016. As the first recombinant treatment for von Willebrand disease (VWD), VONVENDI represents a significant therapeutic advancement in patient care.
“Researchers, clinicians and partners in the global hematology community have set a very high bar for safety and efficacy standards as new treatment options are introduced to address the distinct needs of patients with bleeding disorders,” said Philip J. Vickers, Ph.D., Head of Research and Development, Shire. “Shire is proud to embrace and support these standards with a broad portfolio of factor treatments, a robust pipeline that builds on the proven approach of factor replacement, and our commitment to enhancing holistic patient care, today and in the future.”
Important Information for ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated]
ADYNOVATE, [Antihemophilic Factor (Recombinant), PEGylated], is a human antihemophilic factor indicated in adolescent and adult patients (12 years and older) with hemophilia A (congenital factor VIII deficiency) for:
ADYNOVATE is not indicated for the treatment of von Willebrand disease.
DETAILED IMPORTANT RISK INFORMATION
ADYNOVATE is contraindicated in patients who have had prior anaphylactic reaction to ADYNOVATE, to the parent molecule (ADVATE), mouse or hamster protein, or excipients of ADYNOVATE (e.g. Tris, mannitol, trehalose, glutathione, and/or polysorbate 80).
WARNINGS & PRECAUTIONS
Hypersensitivity reactions are possible with ADYNOVATE. Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with other recombinant antihemophilic factor VIII products, including the parent molecule, ADVATE. Early signs of hypersensitivity reactions that can progress to anaphylaxis may include angioedema, chest tightness, dyspnea, wheezing, urticaria, and pruritus. Immediately discontinue administration and initiate appropriate treatment if hypersensitivity reactions occur.
Formation of neutralizing antibodies (inhibitors) to factor VIII can occur following administration of ADYNOVATE. Monitor patients regularly for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests. Perform an assay that measures factor VIII inhibitor concentration if the plasma factor VIII level fails to increase as expected, or if bleeding is not controlled with expected dose.
Common adverse reactions (≥1% of subjects) reported in the clinical studies were headache and nausea.
For ADYNOVATE Full Prescribing Information, visit: http://baxalta.com/assets/documents/ADYNOVATE_PI.pdf.
Important Information for ADVATE [Antihemophilic Factor (Recombinant)]
ADVATE [Antihemophilic Factor (Recombinant)] is a recombinant antihemophilic factor indicated for use in children and adults with hemophilia A (congenital factor VIII deficiency) for:
ADVATE is not indicated for the treatment of von Willebrand disease.
DETAILED IMPORTANT RISK INFORMATION
ADVATE is contraindicated in patients who have life-threatening hypersensitivity reactions, including anaphylaxis, to mouse or hamster protein or other constituents of the product.
WARNINGS & PRECAUTIONS
Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with ADVATE. Symptoms include dizziness, paresthesia, rash, flushing, facial swelling, urticaria, dyspnea, pruritus, and vomiting.
Discontinue ADVATE if hypersensitivity symptoms occur and administer appropriate emergency treatment.
Neutralizing antibodies (inhibitors) have been reported following administration of ADVATE predominantly in previously untreated patients (PUPs) and previously minimally treated patients (MTPs). Monitor all patients for the development of factor VIII inhibitors by appropriate clinical observation and laboratory testing. If expected plasma factor VIII activity levels are not attained, or if bleeding is not controlled with an expected dose, perform an assay that measures factor VIII inhibitor concentration.
Serious adverse reactions seen with ADVATE are hypersensitivity reactions, including anaphylaxis, and the development of high-titer inhibitors necessitating alternative treatments to factor VIII.
The most common adverse reactions observed in clinical trials (frequency ≥5% of subjects) were pyrexia, headache, cough, nasopharyngitis, arthralgia, vomiting, upper respiratory tract infection, limb injury, nasal congestion, and diarrhea.
For ADVATE Full Prescribing Information, visit: http://www.baxalta.com/assets/documents/ADVATE_PI.pdf.
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
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 VONVENDI Full Prescribing Information, visit: http://www.baxalta.com/assets/documents/VONVENDI_PI.pdf.
FOR FURTHER INFORMATION PLEASE CONTACT:
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NOTES TO EDITORS
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.
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:
other risks and uncertainties detailed from time to time in Shire’s, Dyax’s or Baxalta’s filings with the Securities and Exchange Commission, including those risks outlined in “ITEM 1A: Risk Factors” in Shire’s and Baxalta’s Annual Reports on Form 10-K for the year ended December 31, 2015.
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 republish revised forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events.