The European Commission ( EC ) has approved Alprolix ( rFIXFc ) 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.
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 more than 0.5% 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 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.
This technology enables Alprolix to use a naturally occurring pathway to prolong the time the therapy remains in the body.
Haemophilia B is caused by having substantially reduced or no factor IX activity, which is needed for normal blood clotting.
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.
The World Federation of Haemophilia estimates that approximately 28,000 people are currently diagnosed with haemophilia B worldwide. ( Xagena )
Source: SOBI, 2016