OREANDA-NEWS. 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-H?m). 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]

Indication
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.

Important Information for ADVATE [Antihemophilic Factor (Recombinant)]

Indication
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.