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Date: 2014-12-19

Type of information: Granting of a Market Authorisation in the EU

Product name: Rixubis® - BAX 326

Compound: recombinant factor IX (rFIX - nonacog gamma)

Therapeutic area: Hematological diseases - Genetic diseases - Rare diseases

Action mechanism:

Nonacog gamma is an antihaemorrhagic, blood coagulation factor IX which when activated, in combination with activated factor VIII, activates factor X to convert prothrombin into thrombin; thrombin then converts fibrinogen into fibrin and a clot is formed.

Company: Baxter (USA - IL)

Disease:

treatment and prophylaxis of bleeding episodes for patients over 16 years of age with hemophilia B

routine prophylactic treatment, control and prevention of bleeding episodes, and perioperative management in children with hemophilia B

Latest news:

* On 23 October 2014, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for Rixubis®, 250 IU, 500 IU, 1000 IU, 2000 IU, 3000 IU, powder and solvent for solution for injection intended for treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor IX deficiency).The benefits with Rixubis® are its ability to achieve haemostatic efficacy for the prevention and treatment of bleeds in patients with Haemophilia B as well as efficacy during surgery. The most common side effects are dysgeusia, pain in extremity, anti Furin antibodies and postoperative anaemia. A pharmacovigilance plan for Rixubis® will be implemented as part of the marketing authorisation.

* On September 15, 2014, Baxter International announced that the FDA has approved Rixubis® [Coagulation Factor IX (Recombinant)] for routine prophylactic treatment, control and prevention of bleeding episodes, and perioperative management in children with hemophilia B. Rixubis® was the first recombinant factor IX (rFIX) approved for routine prophylaxis and control of bleeding episodes in the U.S. for adults living with this chronic condition. The approval is based on the results of a clinical trial investigating the efficacy and safety of RIXUBIS among 23 previously-treated male patients less than 12 years of age with severe or moderately severe hemophilia B. The patients were treated with a twice-weekly Rixubis® prophylaxis regimen (mean dose 56 IU/kg) for a mean treatment duration of six months and a mean of 54 exposure days (EDs). The median annualized bleeding rate (ABR) was 2.0 (0.0 for spontaneous bleeds and joint bleeds). Nine patients in the study (39.1%) experienced no bleeds and 23 bleeding episodes (88.5%) were treated with 1-2 infusions. There were no reports of inhibitor development, no severe allergic reactions, and no thrombotic or treatment-related adverse events among the study participants. Common adverse reactions observed in >1% of subjects in clinical studies were dysgeusia, pain in extremity, and positive test for furin antibody. These data were presented during the 55th Annual Meeting of the American Society of Hematology (ASH) in New Orleans, LA. Baxter’s application for marketing approval for Rixubis® for adults and pediatric patients is currently under review in the European Union, with a regulatory decision expected later this year. The treatment also recently gained regulatory approval in Australia.

* On December 9, 2013, Baxter International has announced that it has submitted an application to the FDA for a pediatric indication for Rixubis® to treat hemophilia B. The submission was based on the results of a study presented during the 55th Annual Meeting of the American Society of Hematology (ASH) in New Orleans. The Phase II/III clinical trial investigated the efficacy and safety of Rixubis® among 23 previously-treated male patients less than 12 years of age with severe or moderately severe hemophilia B. The patients were treated with a twice-weekly Rixubis® prophylaxis regimen (median dose 56 IU/kg) over six months or for a minimum of 50 exposure days (EDs). The median annualized bleeding rate (ABR) was 2.0 (0.0 for spontaneous bleeds and joint bleeds). Nine patients in the study (39.1%) experienced no bleeds and 23 patients (88.5%) were treated with 1-2 infusions. In hemostatic efficacy, Rixubis® was rated excellent or good at resolving bleeds in 96 percent of all episodes. Of the 26 bleeds seen in the trial, only two (in two patients) were spontaneous. There were no reports of inhibitor development, no allergic reactions, and no thrombotic or treatment-related adverse events among the study participants.

* On October 31, 2013, Baxter International has announced that the company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for approval of Rixubis®, (nonacog gamma) for the treatment and prophylaxis of bleeding in patients of all ages with hemophilia B. Hemophilia B, also known as Christmas disease, is the second most common type of hemophilia and results from insufficient amounts of clotting factor IX, a naturally occurring protein in blood that helps to control bleeding. 

Rixubis® was approved for adult patients and launched in the United States and Puerto Rico earlier in 2013. The filing is based on results of research with hemophilia B patients previously treated with other therapies. A global Phase I/III study of adult patients with severe or moderately severe hemophilia B demonstrated that twice-weekly prophylactic treatment with  Rixubis® for six months achieved a median annualized bleed rate (ABR) of 2.0, with 43 percent (24/56) of patients experiencing no bleeds. In the study, no patients developed an inhibitory antibody to FIX and no cases of anaphylaxis were reported. The most common adverse reactions observed in >1% of subjects of this study were dysgeusia, and pain in extremity. The application is further supported by data from a study of 23 hemophilia B patients less than twelve years old.

* On June 26, 2013, the FDA has approved first recombinant coagulation factor IX that is specifically indicated for routine use in preventing bleeding episodes (prophylaxis) in people with hemophilia B who are 16 years of age and older. Rixubis® is indicated for the control and prevention of bleeding episodes, perioperative (period extending from the time of hospitalization for surgery to the time of discharge) management, and routine use to prevent or reduce the frequency of bleeding episodes (prophylaxis).The efficacy of Rixubis® was evaluated in a multicenter study in which a total of 73 male patients between 12 and 65 years of age received Rixubis for routine prophylaxis or as needed in response to symptoms of bleeding (on-demand). Overall, patients in the prophylaxis study had a 75 percent lower annual bleeding rate when compared to patients who have historically received on-demand treatment. An additional study in a pediatric population is currently ongoing. Although serious side effects including anaphylaxis (life-threatening allergic reactions) can occur, the most common side effects observed in patients in clinical studies were dysgeusia (distorted taste), pain in an extremity, and atypical blood test results.

* On September 4, 2012, Baxter International has announced that the company has submitted a biologics license application (BLA) to the FDA for approval of BAX 326, a recombinant factor IX (rFIX) protein being investigated for the treatment and prophylaxis of bleeding episodes for patients over 12 years of age with hemophilia B. The BLA filing is based on results from a global Phase III study conducted in 10 countries around the world. The prospective, controlled, multicenter study evaluated the pharmacokinetics, efficacy, safety and immunogenicity of BAX 326 in 73 patients with severe or moderately severe hemophilia B previously treated with other factor IX therapy. The study met its primary objectives and the company plans to present the complete data from the study in late 2012.  Baxter expects to file its application for BAX 326 in Europe in 2013.

 

Patents:

Submission of marketing authorization application USA : 2012-09-04

Submission of marketing authorization application UE: 2013-10-31

Withdrawal of marketing authorization application USA:

Withdrawal of marketing authorization application UE:

US authorization: 2013-06-26/2014-09-15

UE authorization: 2014-12-19

Favourable opinion UE:

Favourable opinion USA:

Orphan status USA:

Orphan status UE:

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes