Shire granted EU marketing authorization for ADYNOVI® [Antihemophilic Factor (Recombinant), PEGylated] for adults and adolescents with Hemophilia A
Zug, Switzerland - January 15, 2018 - Shire plc (LSE: SHP, NASDAQ: SHPG), the global biotechnology leader in rare diseases, announced today that the European Commission (EC) has granted Marketing Authorization for ADYNOVI [Antihemophilic Factor (Recombinant), PEGylated], an extended half-life recombinant factor VIII (rFVIII) treatment, for on-demand and prophylactic use in patients 12 years and older living with hemophilia A. ADYNOVI is modified to last longer in the blood and potentially require less frequent injections than unmodified Antihemophilic Factor when used to reduce the frequency of bleeding. It is built on ADVATE® [Antihemophilic Factor (Recombinant)], a treatment used by hemophilia A patients worldwide for almost 15 years. ADYNOVI's proprietary PEGylation technology, exclusively licensed from Nektar Therapeutics, extends the time between treatments and offers a twice-weekly dosing schedule.1
"The European approval of ADYNOVI is an important milestone in our continued commitment to provide new treatment options for patients living with hemophilia A," said Dr. Peter Foertig, MD, Global Head Hematology Medical Affairs, Shire. "We believe that the twice-weekly prophylactic dosing, as well as the on-demand control of bleeding, offered by ADYNOVI will bring us closer to our goal of improving and personalizing disease management for hemophilia A patients in Europe."
The Marketing Authorization is based on outcomes from three Phase 3 clinical trials of patients with hemophilia A. These include a prospective, global, multi-center, open label, non-randomized study of patients 12 to 65 years of age; a prospective, uncontrolled, open label, multi-center study of patients 12 years of age and younger; and a study of perioperative control of hemostasis with interim study results from 15 patients with severe hemophilia A undergoing surgical procedures.2,3,4
Hemophilia A, designated an orphan disease by the EC, is a rare bleeding disorder that causes longer-than-normal bleeding due to lack of proper clotting factor VIII (FVIII) in the blood.5,6 The severity of hemophilia A is determined by the amount of factor in the blood, with more severity associated with lower amounts of factor.7 More than half of patients with hemophilia A have the severe form of the condition.7 Today, hemophilia A affects approximately 150,000 across the globe.8 It primarily affects males, with an incidence of one in 5,000 male births.9 Of the worldwide hemophilia patient population, an estimated 75 percent lack adequate treatment or access to treatment altogether.9
With this approval, Shire is now authorized to market ADYNOVI in the 28 Member States of the European Union (EU), as well as in Iceland, Liechtenstein and Norway.
ADYNOVI was first approved as ADYNOVATE® [Antihemophilic Factor (Recombinant), PEGylated] by the Food and Drug Administration (FDA) in the U.S. followed by approval in Japan, Canada, and Colombia, and is approved as ADYNOVI in Switzerland. ADYNOVATE most recently received approval in Japan for use in hemophilia A pediatric patients under 12 years of age and those undergoing surgery in November 2017.
ADYNOVI SAFETY INFORMATION FOR EUROPE
Please consult the ADYNOVI Summary of Product Characteristics (SmPC) before prescribing, particularly in relation to dosing and treatment monitoring.
Hypersensitivity to the active substance, to the parent molecule octocog alfa or to any of the excipients listed in the SmPC. Known allergic reaction to mouse or hamster protein.
Special warnings and precautions for use
The medicinal product contains traces of mouse and hamster proteins. If symptoms of hypersensitivity occur, patients should be advised to discontinue use of the medicinal product immediately and contact their physician. Patients should be informed of the early signs of hypersensitivity reactions including hives, generalised urticaria, tightness of the chest, wheezing, hypotension, and anaphylaxis.
The formation of neutralising antibodies (inhibitors) against factor VIII is a known complication in the management of individuals with haemophilia A. These inhibitors are usually IgG immunoglobulins directed against the factor VIII procoagulant activity, which are quantified in Bethesda Units (BU) per ml of plasma using the modified assay.
In general, all patients treated with coagulation factor VIII should be carefully monitored for the development of inhibitors by appropriate clinical observations and laboratory tests. If the expected factor VIII activity plasma levels are not attained, or if bleeding is not controlled with an appropriate dose, testing for factor VIII inhibitor presence should be performed.
After reconstitution this medicinal product contains 0.45 mmol sodium (10 mg) per vial.
|Common (Greater-than or equal to 1/100 to <1/10)||Headache, Diarrhea, Nausea, Rash|
|Uncommon (Greater-than or equal to 1/1000 to <1/100)||Factor VIII inhibition in previously-treated patients (PTPs), Hypersensitivity, Flushing|
For more information, please refer to the ADYNOVI Summary of Product Characteristics here.
ADVATE SAFETY INFORMATION FOR EUROPE
ADVATE is contraindicated in patients with known anaphylaxis to mouse or hamster protein or other constituents of the product.
Allergic-type hypersensitivity reactions, including anaphylaxis, are possible and have been reported with ADVATE. Symptoms have manifested as dizziness, paresthesia, rash, flushing, face swelling, urticaria, dyspnea, and pruritus. Discontinue use if hypersensitivity symptoms occur and administer appropriate emergency treatment.
Carefully monitor patients treated with factor VIII products for the development of FVIII inhibitors by appropriate clinical observations and laboratory tests. Inhibitors have been reported following administration of ADVATE predominantly in previously untreated patients (PUPs) and previously minimally treated patients (MTPs).
If expected plasma FVIII levels are not attained, or if bleeding is not controlled with an expected dose, perform an assay that measures FVIII inhibitor concentration.
The serious adverse reactions seen with ADVATE are hypersensitivity reactions and the development of high-titer inhibitors necessitating alternative treatments to FVIII.
The most common adverse reactions observed in clinical trials (frequency greater than or equal to 10 percent of subjects) were pyrexia, headache, cough, nasopharyngitis, vomiting, arthralgia, and limb injury.
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NOTES TO EDITORS
Shire is the global leader in serving patients with rare diseases. We strive to develop best-in-class therapies across a core of rare disease areas including hematology, immunology, genetic diseases, neuroscience, and internal medicine with growing therapeutic areas in ophthalmics and oncology. Our diversified capabilities enable us to reach patients in more than 100 countries who are struggling to live their lives to the fullest.
We feel a strong sense of urgency to address unmet medical needs and work tirelessly to improve people's lives with medicines that have a meaningful impact on patients and all who support them on their journey.
Statements included herein that are not historical facts, including without limitation statements concerning future strategy, plans, objectives, expectations and intentions, projected revenues, 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:
a further list and description of risks, uncertainties and other matters can be found in Shire's most recent Annual Report on Form 10-K and in Shire's subsequent Quarterly Reports on Form 10-Q, in each case including those risks outlined in "ITEM 1A: Risk Factors", and in Shire's subsequent reports on Form 8-K and other Securities and Exchange Commission filings, all of which are available on Shire's website.
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