Swedish Orphan Biovitrum AB (publ) (Sobi), an international specialty healthcare company and Biogen, one of the world’s oldest independent biotechnology companies, announced that the European Commission (EC) has approved Alprolix (rFIXFc), their extended half-life therapy, for the treatment of haemophilia B in all 28 European Union (EU) member states and maintained its orphan designation. Alprolix is the only recombinant factor IX Fc Fusion protein therapy for haemophilia B to offer people in the EU prolonged protection against bleeding episodes with fewer prophylactic injections.
Alprolix is indicated for both on-demand and prophylaxis treatment of people with haemophilia B in all ages. Prophylactically, it can be administered with an initial dose every seven days or every 10 days with the ability to adjust the dosing interval based on individual response.
“With the approval of Alprolix, people with haemophilia B in Europe now have the potential to experience prolonged protection from bleeds with fewer injections,” said Krassimir Mitchev, M.D., Ph.D., vice president and medical therapeutic area head of haemophilia at Sobi. “We are working to make Alprolix available in Europe as quickly as possible. Along with Biogen, we’re excited to continue offering innovative therapies to people with haemophilia around the world.”
The EC’s approval of Alprolix was based on results from two global phase 3 clinical trials that demonstrated the efficacy, safety and pharmacokinetics of Alprolix for haemophilia B: the pivotal B-LONG study for previously treated adults and adolescents, and the Kids B-LONG study for previously treated children under age 12. The adverse drug reactions with an incidence of = 0.5 percent for Alprolix were nasopharyngitis (common cold), influenza, arthralgia (joint pain), upper respiratory tract infection, headache, and hypertension. The majority of these events were judged as not related or likely not related to study drug.
“Alprolix has become a meaningful treatment advance for people living with haemophilia B in countries where it is approved and is backed by robust clinical data and the longest real-world experience of any prolonged circulation factor IX therapy to date,” said Gilmore O’Neill, M.D., senior vice president Drug Innovation Units at Biogen. “We’re proud to bring the European haemophilia community one of the first treatment advances in nearly 20 years, and believe the availability of extended half-life therapies in Europe will change the way that many approach treatment.”
Sobi and Biogen collaborate on the development and commercialisation of Alprolix for haemophilia B. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia and most Middle Eastern markets). Biogen leads development and manufacturing for Alprolix and has commercialisation rights in North America and all other regions in the world excluding the Sobi territory.
Alprolix is a recombinant clotting factor therapy developed for haemophilia B by fusing factor IX to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body). This technology enables Alprolix to use a naturally occurring pathway to prolong the time the therapy remains in the body. While Fc fusion has been used for more than 15 years, Sobi and Biogen are the first companies to utilise it in the treatment of haemophilia.
Alprolix is currently approved for the treatment of haemophilia B in the United States, European Union, Canada, Japan, Australia, New Zealand, and other countries, to provide prolonged protection from bleeds. As with any infused protein, allergic type hypersensitivity reactions and development of inhibitors may occur following administration of Alprolix.
Haemophilia B is caused by having substantially reduced or no factor IX activity, which is needed for normal blood clotting. The World Federation of Haemophilia estimates that approximately 28,000 people are currently diagnosed with haemophilia B worldwide.
People with haemophilia B may experience bleeding episodes in joints and muscles that cause pain, decreased mobility and irreversible joint damage. In the worst cases, these bleeding episodes can cause organ bleeds and life-threatening hemorrhages. Infusions of factor IX temporarily replace clotting factors necessary to resolve bleeding and, when used prophylactically, to prevent new bleeding episodes.