Swedish Orphan Biovitrum AB (publ) (Sobi) (STO: SOBI) and Biogen (NASDAQ: BIIB) have announced that the European Commission (EC) has approved Elocta® (rFVIIIFc) for the treatment of haemophilia A in all 28 European Union (EU) member states, as well as Iceland, Liechtenstein and Norway. Elocta, a recombinant factor VIII Fc fusion protein with an extended half-life, will be the first haemophilia A treatment in the EU to offer prolonged protection against bleeding episodes with prophylactic injections every three to five days. The product will be available in the first EU countries, which includes the UK in early 2016.
"The EC's approval of Elocta is an important milestone for Sobi, and we hope, the haemophilia community in the UK & Ireland, offering the potential to improve the care of people with haemophilia A," said Neil Dugdale, General Manager Sobi, UK & Ireland. "Our focus is now to ensure timely and sustainable access to Elocta for people living with haemophilia A."
Elocta is indicated for both on-demand and prophylaxis treatment of people with haemophilia A of all ages. The EC approval was based on data from Elocta's pivotal, phase 3 A-LONG clinical study, which demonstrated the efficacy, safety and pharmacokinetics of rFVIIIFc in previously treated males 12 years of age and older with severe haemophilia A, and from the phase 3 Kids A-LONG clinical study, which demonstrated the efficacy and safety of rFVIIIFc in previously treated male children with haemophilia A under 12 years of age.
John Pasi, Professor of Haemostasis and Thrombosis, at Barts and the London, Queen Mary, Univeristy of London said, "This signifies a real milestone for haemophilia care in UK and Ireland - the beginning of the next generation of therapy for haemophilia A. We can potentially improve care for our patients, both protecting against bleeding and also tailoring treatment to the individual. As a clinician it's hugely encouraging to see such positive developments in the treatment options we have at our disposal."
"A recent survey of our members highlighted fewer joint problems, less pain and fewer bleeds as the top three factors people with haemophilia feel would improve their quality of life," said Liz Carroll, Chief Executive of The UK Haemophilia Society. "We hope that this new treatment option will be part of helping to make Haemophilia a smaller and smaller part of some families lives."
Sobi and Biogen are collaboration partners in the development and commercialisation of rFVIIIFc for haemophilia A. Last year, Sobi exercised its opt-in right to assume rFVIIIFc's final development and commercialisation in pre-specified territories, which essentially include Europe, North Africa, Russia and certain countries in the Middle East. Biogen leads development and manufacturing of the product and holds commercialisation rights in North America and all other regions in the world outside of the Sobi territory.
Elocta is the trade name for rFVIIIFc in Sobi's territory, which is also approved under the name Eloctate® [Antihemophilic Factor (Recombinant), Fc Fusion Protein] for the treatment of haemophilia A in the U.S., Canada, Australia, New Zealand and Japan.
About Elocta®
Elocta (rFVIIIFc) is the first recombinant clotting factor VIII therapy that offers an extended half-life in the body. It is indicated for the treatment and prophylaxis of bleeding episodes in patients with haemophilia A (factor VIII deficiency) and can be used by people of all ages. Elocta was developed by fusing B-domain deleted factor VIII to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body). It is believed that this enables Elocta to utilise a naturally occurring pathway to prolong the time the therapy remains in the body. While Fc fusion technology has been used in other therapies for more than 15 years, Sobi and Biogen are the first companies to utilise it in the treatment of haemophilia. Allergic type hypersensitivity reactions and development of Factor VIII neutralising antibodies (inhibitors) may occur following administration of Elocta.
About Haemophilia A
Haemophilia A is a rare, chronic, genetic disorder in which the ability of a person's blood to clot is impaired due to missing or reduced levels of a protein known as factor VIII. People with haemophilia A experience bleeding episodes that may cause pain, irreversible joint damage and life-threatening haemorrhages. According to the World Federation of Haemophilia, an estimated 140,000 people worldwide are identified as living with haemophilia A.
Therapies for haemophilia A, the most common form of haemophilia, can be administered either on a schedule to help prevent or reduce bleeding episodes (prophylaxis) or to control bleeding when it occurs (on-demand). The World Federation of Hemophilia recommends that prophylaxis be the goal of therapy because it may prevent bleeding and joint destruction. As a result, regular prophylactic treatment may slow progression of joint disease and may improve quality of life.