Biogen and Swedish Orphan Biovitrum AB (publ) (Sobi), an international specialty healthcare company dedicated to rare diseases,  announced that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) of Alprolix (rFIXFc), a recombinant factor IX Fc fusion protein product candidate for the treatment of haemophilia B. This validation signifies the initiation of the EMA’s review process.

The MAA includes results from two global, phase 3 clinical trials examining the efficacy, safety and pharmacokinetics (a measure of the presence of the therapy in a person’s body over time) of Alprolix for haemophilia B: the pivotal B-LONG study for previously treated adults and adolescents, and Kids B-LONG study for previously treated children under age 12.

Alprolix is a recombinant, clotting factor IX therapy and is currently approved for the treatment of haemophilia B in the US, Canada, Japan and Australia. It is the only approved haemophilia B therapy to demonstrate prolonged clotting factor circulation in the body.

“The acceptance of this MAA is an important milestone in our goal to bring this innovative therapy to the European haemophilia community,” said Douglas E. Williams, Ph.D., executive vice president of research and development at Biogen. “We look forward to working with European regulators to help people with haemophilia B in Europe realise the benefits that treatment with Alprolix may offer.”

Biogen and Sobi are collaborators in the development and commercialisation of Alprolix for haemophilia B. Sobi has an opt-in right to assume final development and commercialisation of Alprolix for the Sobi territories (essentially, Europe, North Africa, Russia and certain Middle Eastern markets). Biogen leads development for Alprolix, has manufacturing rights, and has commercialisation rights in North America and all other regions in the world excluding the Sobi territories.

“Our collaboration with Biogen remains focused on the goal of transforming the care of people living with haemophilia,” said Birgitte Volck, M.D., Ph.D., senior vice president of development and chief medical officer of Sobi. “The validation of the Alprolix MAA marks a critical regulatory milestone in our continued, collaborative global efforts.”

The World Federation of Haemophilia global survey conducted in 2013 estimates that approximately 28,430 people are currently diagnosed with haemophilia B worldwide. It is caused by having substantially reduced or no factor IX activity, which is needed for normal blood clotting. People with haemophilia B experience bleeding episodes that cause pain, irreversible joint damage, and life-threatening haemorrhages. Prophylactic infusions of factor IX temporarily replace clotting factors necessary to control bleeding and prevent new bleeding episodes.

B-LONG was a global, open-label, multi-center phase 3 study that evaluated the efficacy, safety and pharmacokinetics of Alprolix in 123 males aged 12 years and older with severe haemophilia B and a history of at least 100 exposure days on any currently available factor IX therapy. The study involved 50 haemophilia treatment centres in 17 countries on six continents. It examined the effect of Alprolix for prophylaxis, episodic (on-demand) treatment, and during surgery (perioperative management). Starting prophylaxis regimens were either 50 IU/kg once weekly or 100 IU/kg every 10 days. The dose or interval could be adjusted as clinically indicated.

Kids B-LONG was a global, open-label, multicenter phase 3 study involving 30 boys under age 12 with severe haemophilia B and at least 50 prior exposure days to factor IX therapies.

Participants in both the B-LONG and Kids B-LONG clinical trials were able to enroll in BYOND, a long-term extension clinical study evaluating the safety and efficacy of Alprolix, which is currently ongoing.

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). It is believed that 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, Biogen is the only company to apply it in haemophilia.

Common adverse reactions (incidence of greater than or equal to 1 per cent) from the B-LONG study were headache and oral paresthesia (an abnormal sensation in the mouth).

Biogen discovers, develops and delivers to patients worldwide innovative therapies for the treatment of neurodegenerative diseases, haematologic conditions and autoimmune disorders.