The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorization for nonacog gamma (Rixubis, Baxter Innovations GmbH) for prophylaxis and treatment of bleeding in patients with hemophilia B, according to an EMA statement.
Nonacog gamma is given by injection in doses of 250, 500, 1000, 2000, and 3000 IU for prevention and treatment of bleeding in patients with hemophilia B (congenital factor IX deficiency). It is recommended for patients of all ages. Its active substance is nonacog gamma, an antihemorrhagic, blood coagulation factor IX (ATC code: B02BD04). When activated, it works with activated factor VII to stimulate factor X to convert prothrombin into thrombin, which in turn converts fibrinogen into fibrin to form a clot. Nonacog gamma is also efficacious during surgery.
The decision follows a review of data from a global phase 1/3 study of adults with severe or moderately severe hemophilia B who were treated prophylactically with nonacog gamma for 6 months. Patients experienced a median annualized bleed rate of 2.0, and 43% (24/56) of patients had no bleeds. None of the patients developed an inhibitory antibody to factor IX and there were no reports of anaphylaxis. The most commonly reported adverse events seen in more than 1% were dysgeusia and extremity pain.
The panel considered additional supportive data from a trial of 23 patients with hemophilia B who were aged less than 12 years.
The US Food and Drug Administration approved recombinant coagulation factor IX for prophylaxis of bleeding in patients aged 16 years or older with hemophilia B in June 2013.
The company will implement a pharmacovigilance plan as a component of the marketing authorization.
Only physicians experienced in the treatment of patients with hemophilia should prescribe nonacog gamma (recombinant). Detailed recommendations for its use will be explained in the summary of product characteristics, which will be published in all official European Union languages in the European public assessment report after the European Commission grants marketing authorization.
The CHMP considers the benefit-to-risk balance for nonacog gamma to be favorable.
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Cite this: Rixubis Recommended by EMA Panel for Hemophilia B - Medscape - Oct 27, 2014.