CHMP recommends Idelvion (albutrepenonacog alfa) for prophylaxis of bleeding in Haemophilia B patients- CSL Behring

The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Idelvion (albutrepenonacog alfa) from CSL Behring GmbH.and intended for treatment and prophylaxis of bleeding in patients with Haemophilia B. Idelvion was designated as an orphan medicinal product on 04 February 2010. Idelvion will be available as 250 IU, 500 IU, 1000 IU and 2000 IU Powder and solvent for solution for injection. The active substance of Ide is albutrepenonacog alfa, an antihaemorrhagic, blood coagulation factor IX, (ATC code: B02BD04). It works as replacement therapy and temporarily increases plasma levels of factor IX, helping to prevent and control bleeding. The benefits with Idelvion are its ability to stop the bleeding when given on demand and prevent bleeding when used as routine prophylaxis or for surgical procedures. The most common side effects are injection site reaction and headache.

Comment: The competitive landscape of hemophilia A and B recombinant therapy in the US and EU is dominated by the use of recombinant FVIII and FIX replacement factors, and patients with severe forms of the disease receive frequent prophylactic infusions beginning from the first one or two years of life and often continuing through adulthood. However, the burden on patients and their families to maintain the prophylactic treatment schedule is high, and there is a significant unmet need for new therapies that can reduce the number of weekly prophylactic infusions and alleviate some of this treatment burden. CSL Behring's CSL-654 ( now Idelvion)is a long-lasting recombinant FIX-albumin fusion protein for on-demand and prophylactic therapy of adults and children with hemophilia B.