On November 4, 2016 Shire plc (LSE: SHP, NASDAQ: SHPG), the leading global biotechnology company focused on serving individuals with rare diseases, reported the depth and breadth of the data it will be presenting at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, taking place December 3-6 in San Diego, California (Press release, Shire, NOV 4, 2016, View Source [SID1234516323]). The company’s robust research in hematology, oncology and genetic disease will be showcased in 4 oral presentations and 12 poster presentations.
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"Shire’s extensive presence at ASH (Free ASH Whitepaper) 2016 reinforces our commitment to discovering and delivering transformative treatments for patients with rare conditions," said Philip J. Vickers, Ph.D., Head of Research and Development, Shire. "We aim to continually lead the scientific dialogue for rare hematologic research and address some of the most urgent unmet needs for patients facing these challenging diseases."
HEMATOLOGY
Data in hematology, the largest portfolio for the company, will focus on research that continues to build on the proven approach of direct factor replacement as the global standard of care for hemophilia. Data presentations will showcase the continued growth of the portfolio and new strategies to improve outcomes for people with hemophilia and other bleeding conditions. The company will also present research on several of its promising early- and late-stage pipeline programs:
Target Joint Status in Patients with Hemophilia A During 18 Consecutive Months of Prophylaxis with a Pegylated Full-Length Recombinant Factor VIII with Extended Half-Life Pub #2592. Session #322. Session Title: Disorders of Coagulation or Fibrinolysis. Poster # II. Sunday, Dec. 4, 2016 6 p.m. – 8 p.m. in Hall GH (San Diego Convention Center)
Modelling FVIII Levels for Prediction of Zero Spontaneous-Joint Bleeding in a Cohort of Severe Hemophilia A Subjects with Target Joints Initiated on Tertiary Prophylaxis Pub #2576. Session #322. Session Title: Disorders of Coagulation or Fibrinolysis. Poster # II. Sunday, Dec. 4, 2016 6 p.m. – 8 p.m. in Hall GH (San Diego Convention Center)
Pharmacodynamic Profile of a Recombinant ADAMTS13 (BAX930) in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome (USS)) Pub #135. Session #311. Session Title: Disorders of Platelet Number of Function: TTP and HUS. Saturday, Dec. 3, 2016 12 p.m. – 1:30 p.m. in Room 29 (San Diego Convention Center)
Appearance of High-Affinity Antibodies Precedes Clinical Diagnosis of FVIII Inhibitors – Preliminary Analysis from the Hemophilia Inhibitor PUP Study (HIPS) Pub #328. Session #322. Session Title: Disorders in Coagulation or Fibrinolysis: Hemophilia Inhibitors. Sunday, Dec. 4, 2016 9:30 a.m. – 11 a.m. in Room 33 (San Diego Convention Center)
ONCOLOGY
As part of Shire’s focus on serving individuals with rare diseases, the company is committed to addressing unmet needs in areas of oncology where patients and providers have limited treatment options. Shire has pipeline assets and research targets for rare cancers including metastatic pancreatic cancer, colorectal cancer and targets in checkpoint inhibitors and allogeneic CAR-T, as well as a biologic treatment approved as part of a multi-agent chemotherapeutic regimen for the treatment of acute lymphocytic leukemia (ALL), ONCASPARTM (pegaspargase), that is approved by the U.S. Food & Drug Administration (FDA) and all 27 EU member states. Data presented at ASH (Free ASH Whitepaper) will include a focus on pegaspargase as a therapeutic option for ALL.
GENETIC DISEASE
Shire is also dedicated to helping patients with inherited illnesses. Type I Gaucher disease is a rare, inherited metabolic condition, and the most common of a family of rare diseases known as lysosomal storage disorders (LSDs). It affects approximately 1 in 50,000 to 1 in 100,000 people in the general population, and 1 in 855 people in the Ashkenazi Jewish community. Every type I Gaucher patient is unique and will experience varying symptoms and degrees of disease severity, making type I Gaucher disease difficult to diagnose. To this end, Shire will be hosting an engaging, case-based presentation to raise disease awareness:
Product Theater: Julie M: A Diagnostic Journey. Monday, Dec. 5, 12:15 p.m. to 1:15 p.m.
Important Information for ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated]
Indications
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:
On-demand treatment and control of bleeding episodes
Routine prophylaxis to reduce the frequency of bleeding episodes
ADYNOVATE is not indicated for the treatment of von Willebrand disease.
DETAILED IMPORTANT RISK INFORMATION
CONTRAINDICATIONS
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
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.
Neutralizing Antibodies
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.
ADVERSE REACTIONS
Common adverse reactions (≥1% of subjects) reported in the clinical studies were headache and nausea.
For Full Prescribing Information, visit http://www.shirecontent.com/PI/PDFs/ADYNOVATE_USA_ENG.pdf
Important Information for ADVATE [Antihemophilic Factor (Recombinant)]
Indications
ADVATE [Antihemophilic Factor (Recombinant)] is a recombinant antihemophilic factor indicated for use in children and adults with hemophilia A (congenital factor VIII deficiency) for:
Control and prevention of bleeding episodes
Perioperative management
Routine prophylaxis to prevent or reduce the frequency of bleeding episodes
ADVATE is not indicated for the treatment of von Willebrand disease.
DETAILED IMPORTANT RISK INFORMATION
CONTRAINDICATIONS
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
Hypersensitivity Reactions
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
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.
ADVERSE REACTIONS
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 Full Prescribing Information, visit http://www.shirecontent.com/PI/PDFs/ADVATE_USA_ENG.pdf
Important Information for ONCASPARTM (pegaspargase)
Indications
ONCASPAR is an asparagine specific enzyme indicated as a component of a multi-agent chemotherapeutic regimen for treatment of patients with:
First line acute lymphoblastic leukemia
Acute lymphoblastic leukemia and hypersensitivity to asparaginase
DETAILED IMPORTANT RISK INFORMATION
CONTRAINDICATIONS
ONCASPAR is contraindicated in patients who have:
History of serious allergic reactions to ONCASPAR
History of serious thrombosis with prior L-asparaginase therapy
History of pancreatitis with prior L-asparaginase therapy
History of serious hemorrhagic events with prior L-asparaginase therapy
WARNINGS & PRECAUTIONS
Anaphylaxis and Serious Reactions
Anaphylaxis and serious allergic reactions can occur therefore, patients should be observed for one hour after administration. Discontinue ONCASPAR in patients with serious allergic reactions.
Thrombosis
Serious thrombotic events, including sagittal sinus thrombosis can occur Discontinue ONCASPAR in patients with serious thrombotic events.
Pancreatitis
Pancreatitis can occur. Evaluate patients with abdominal pain for evidence of pancreatitis. Discontinue ONCASPAR in patients with pancreatitis.
Glucose Intolerance
Glucose intolerance can occur. In some cases, glucose intolerance is irreversible. Monitor serum glucose.
Coagulopathy
Increased prothrombin time, increased partial thromboplastin time, and hypofibrinogenemia can occur. Monitor coagulation parameters at baseline and periodically during and after treatment.
Hepatotoxicity and Abnormal Liver Function
Hepatotoxicity and abnormal liver function can occur. Perform appropriate monitoring.
ADVERSE REACTIONS
Most common adverse reactions (≥2%) are allergic reactions (including anaphylaxis), hyperglycemia, pancreatitis, central nervous system (CNS) thrombosis, coagulopathy, hyperbilirubinemia, and elevated transaminases.
Hyperlipidemia (hypercholesterolemia and hypertriglyceridemia) has been reported in patients exposed to ONCASPAR.
For Full Prescribing Information, visit http://www.shirecontent.com/PI/PDFs/ONCASPAR_USA_ENG.pdf
Important Information for VPRIV (velaglucerase alfa for injection)
Indications
VPRIV is a hydrolytic lysosomal glucocerebroside-specifi enzyme indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.
DETAILED IMPORTANT RISK INFORMATION
CONTRAINDICATIONS
None
WARNINGS & PRECAUTIONS
Hypersensitivity Reactions
Hypersensitivity reactions, including anaphylaxis, have occurred with VPRIV. The most commonly observed symptoms of hypersensitivity reactions were headache, dizziness, hypotension, hypertension, nausea, fatigue/asthenia, and pyrexia/body temperature increased. Additional hypersensitivity reactions of chest discomfort, dyspnea, and pruritus have been reported with VPRIV. As with any intravenous protein product, hypersensitivity reactions are possible, therefore appropriate medical support including personnel adequately trained in cardiopulmonary resuscitative measures and access to emergency measures should be readily available when VPRIV is administered. If anaphylactic or other acute reactions occur, immediately discontinue the infusion of VPRIV and initiate appropriate medical treatment. Discontinue VPRIV if hypersensitivity symptoms occur.
ADVERSE REACTIONS
Most common adverse reactions during clinical studies in ≥10% of patients were hypersensitivity reactions, headache, dizziness, abdominal pain, nausea, back pain, joint pain, prolonged activated PTT, fatigue/asthenia, and pyrexia.
For Full Prescribing Information, visit http://pi.shirecontent.com/PI/PDFs/Vpriv_USA_ENG.pdf