Swedish Orphan Biovitrum AB (publ) (Sobi), an international speciality healthcare company, and Biogen, one of the world’s oldest independent biotechnology companies, received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending that marketing authorisation be granted for Alprolix (rFIXFc), a recombinant factor IX Fc fusion protein therapy for the treatment of haemophilia B. If approved, Alprolix would be among the first therapies in the European Union (EU) to offer people living with haemophilia B prolonged protection against bleeding episodes with prophylactic dosing intervals.

“This positive opinion marks an important step in our efforts to bring treatment innovation to people with hemophilia in Europe and around the world,” said Krassimir Mitchev, M.D., Ph.D., vice president and medical therapeutic area head of haemophilia at Sobi. “We are already seeing the benefits that Fc fusion technology can offer through our recent EU launch of Elocta for people with haemophilia A. We are excited at the prospect of also offering the possibility for prolonged protection and reduced treatment burden to the haemophilia B community with Alprolix.”

The positive opinion 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 CHMP's recommendation is now referred to the European Commission (EC), which grants marketing authorisation for medicines in the EU.

“Therapies that offer prolonged protection from bleeds are changing the way many approach treatment of haemophilia,” said Gilmore O’Neill, M.D., senior vice president, drug innovation units at Biogen. “We are proud to work with Sobi to continue bringing to Europe these innovative Fc fusion therapies, which are grounded in the most robust real-world experience of any prolonged circulation factor therapies to date.”

Sobi and Biogen are collaborators in 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.

Haemophilia B is caused by having substantially reduced or no factor IX activity, which is needed for normal blood clotting. The World Federation of Hemophilia 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.

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 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 US, Canada, Japan, Australia and New Zealand, where it was the first haemophilia B therapy approved 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.