Products

Date: 2016-09-23

Type of information: Reimbursement

Product name: Eloctate™ (USA)/ Elocta™ (EU)

Compound: recombinant factor VIII Fc fusion protein (rFVIIIFc) - efmoroctocog alfa

Therapeutic area: Eloctate™ (USA)/ Elocta™ (EU)

Action mechanism:

• protein/coagulation factor. Recombinant FVIIIFc/efmoroctocog alfa is a clotting factor developed using Biogen Idec’s novel and proprietary monomeric Fc fusion technology, which makes use of a naturally occurring pathway that delays the destruction of factor and cycles it back into the bloodstream, resulting in a longer circulating half-life.  With this technology, rFVIIIFc is designed to provide long-lasting protection from bleeding and reduce the treatment burden associated with hemophilia A, which currently can require approximately 150 to 180 injections annually for prophylaxis with commercially available factor VIII products.
• Biogen Idec and Swedish Orphan Biovitrum (Sobi) are partners in the development and commercialization of rFIXFc in hemophilia B and rFVIIIFc in hemophilia A. Biogen Idec leads development, has manufacturing rights, and has commercialization rights in North America and all other regions excluding the Sobi territory. Sobi has an opt-in right to take over final development and commercialization of Elocta™ for the Sobi territories (Europe, North Africa, Russia and most Middle Eastern markets). Biogen Idec leads development for Eloctate™/ Elocta™, has manufacturing rights, and has commercialization rights in North America and all other regions in the world excluding the Sobi territories.

Company: BiogenIdec (USA - MA) Swedish Orphan Biovitrum -SOBI (Sweden)

Disease: hemophilia A

Latest news:

• • On September 23, 2016, Swedish Orphan Biovitrum  (Sobi™) announced that Elocta® (efmoroctocog alfa), a recombinant human factor VIII Fc-fusion protein with an extended half-life for the treatment of haemophilia A, has received national reimbursement approval in Spain. Elocta® is already available in the UK, France, Italy, Germany, Sweden, Denmark, Norway, Switzerland, the Netherlands, Slovenia and the Republic of Ireland.
• • On September 21, 2016, Sobi™ announced that Elocta® (efmoroctocog alfa) has been approved for reimbursement in the UK, Italy and France. These countries join Germany, Sweden, Denmark, Norway, Switzerland, the Netherlands, Slovenia and the Republic of Ireland where Eloct®a is already available. This expansion in availability is supported by global experience. With more than two years of post-authorisation real-world experience with Elocta® (marketed as Eloctate® in the US and other regions), over 2,700 patients have been treated in countries where Elocta is commercially available, corresponding to about 1,800 patient-years of experience.
• • On June 27, 2016, Sobi™ announced that the Swiss Agency for Therapeutic Products, Swissmedic, has approved Elocta® (rFVIIIFc) for the treatment of haemophilia A. Elocta® is indicated for both on-demand and prophylaxis treatment of people with haemophilia A of all ages. The Swiss approval was based on data from Elocta®’s pivotal, phase 3 A-LONG clinical study, which demonstrated the efficacy, safety and pharmacokinetics of rFVIIIFc in previously treated males 12 years of age and older with severe haemophilia A, and from the phase 3 Kids A-LONG clinical study, which demonstrated the efficacy and safety of rFVIIIFc in previously treated male children with haemophilia A under 12 years of age.
• • On March 24, 2016, Swedish Orphan Biovitrum announced that the European Commission has approved the transfer of the marketing authorisation for Elocta® (efmoroctocog alfa) from Biogen to Sobi, making Sobi the marketing authorisation holder (MAH) of Elocta® in the EU. As MAH Sobi will assume full legal responsibility for Elocta®, from a regulatory perspective, during its entire life cycle. Sobi and Biogen are collaboration partners in the development and commercialisation of Elocta® for haemophilia A. Sobi holds final development and commercialisation rights in a pre-specified territory, which includes Europe, North Africa, Russia and certain countries in the Middle East. Biogen leads development and manufacturing of the product and holds commercialisation rights in North America and all other regions in the world outside of the Sobi territory.
• • On January 11, 2016, Swedish Orphan Biovitrum announced the commercial launch of Elocta® (efmoroctocog alfa) in first countries in Europe. Elocta® is indicated for both prophylaxis and on-demand treatment of bleeding in people with haemophilia A and can be used for all age groups. The SmPC recommended prophylactic dose of Elocta® is 50 IU/kg every three to five days. The dose may be personalised by the treating physician in the range of 25 to 65 IU/kg depending on the severity of the factor VIII deficiency, the location and frequency of bleeding, and the patient's activity level and clinical condition.
• •  On November 24, 2015, Swedish Orphan Biovitrum and Biogen announced that the European Commission (EC) has approved Elocta® (rFVIIIFc) for the treatment of haemophilia A in all 28 European Union (EU) member states, as well as Iceland, Liechtenstein and Norway. Elocta®, a recombinant factor VIII Fc fusion protein with an extended half-life, will be the first haemophilia A treatment in the EU to offer prolonged protection against bleeding episodes with prophylactic injections every three to five days. Elocta® is indicated for both on-demand and prophylaxis treatment of people with haemophilia A of all ages. The EC approval was based on data from Elocta® ’s pivotal, phase 3 A-LONG clinical study, which demonstrated the efficacy, safety and pharmacokinetics of rFVIIIFc in previously treated males 12 years of age and older with severe haemophilia A, and from the phase 3 Kids A-LONG clinical study, which demonstrated the efficacy and safety of rFVIIIFc in previously treated male children with haemophilia A under 12 years of age. The adverse drug reactions with an incidence of ? 0.5 percent for Elocta® were arthralgia, malaise, myalgia, headache and rash.
• • On September 24, 2015, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for Elocta® (efmoroctocog alfa), intended for the treatment and prophylaxis of bleeding in haemophilia A (congenital factor VIII deficiency). Elocta® was designated as an orphan medicinal product on 20 September 2010. It works as a replacement therapy to increase temporarily the plasma levels of factor VIII, so that the patient is less prone to bleeding and when used on demand or for surgical procedures, to control the bleeding. The benefits with Elocta® are its ability to provide adequate prophylaxis in terms of annualised bleeding rate, to control bleeding on demand and to provide haemostatic efficacy for surgical procedures as seen in clinical trials in adult and paediatric patients. Hypersensitivity reactions have been reported rarely.
• • On April 14, 2015, Sobi announced that the company has received orphan drug designation in Switzerland for Elocta® (rFVIIIFc) developed for the treatment of haemophilia A.
• • On 31 October 2014, Sobi and Biogen Idec announced that the European Medicines Agency (EMA) validated the Marketing Authorisation Application (MAA) for Elocta® (rFVIIIFc), a recombinant factor VIII Fc fusion protein product candidate for the treatment of hemophilia A. The validation of the MAA initiates the EMA’s review process. The regulatory application included results from the pivotal, Phase 3 clinical study, A-LONG that examined the efficacy, safety and pharmacokinetics of rFVIIIFc in males 12 years of age and older with severe hemophilia A and from the Phase 3 clinical study, Kids A-LONG that evaluated the efficacy and safety of rFVIIIFc in children with hemophilia A under 12 years of age.
• • On October 9, 2014, Swedish Orphan Biovitrum announced that Biogen Idec has submitted a Marketing Authorisation Application (MAA) for Elocta® (rFVIIIFc) to the European Medicines Agency (EMA). Sobi has an exclusive opt-in right to assume final development and commercialisation of Elocta in Europe, Russia, the Middle East and Northern Africa. The MAA filing with the EMA triggers the formal opt-in right, giving Sobi approximately two months to exercise its option.
• • On June 6, 2014, the FDA approved Eloctate®, antihemophilic factor, Fc fusion protein, for use in adults and children who have hemophilia A. Eloctate® is the first hemophilia A treatment designed to require less frequent injections when used to prevent or reduce the frequency of bleeding. Eloctate® is approved to help control and prevent bleeding episodes, manage bleeding during surgical procedures, and prevent or reduce the frequency of bleeding episodes (prophylaxis). The safety and efficacy of Eloctate® were evaluated in a clinical trial of 164 patients that compared the prophylactic treatment regimen to on-demand therapy. The trial demonstrated that Eloctate was effective in the treatment of bleeding episodes, in preventing or reducing bleeding and in the control of bleeding during and after surgical procedures. No safety concerns were identified in the trial.
• • On November 12, 2013, Swedish Orphan Biovitrum' (Sobi) partner Biogen Idec, at the Credit Suisse Global Healthcare Conference in Phoenix, has stated that the FDA has requested additional information pertaining to process validation of certain steps in the manufacturing process for Eloctate™.  As a result, Biogen Idec expects a PDUFA extension for Eloctate™ and the company is now planning for a mid-2014 commercial launch in the US based on on-going discussions with the FDA. Sobi believes that this updated timeline for FDA approval is unlikely to impact planned European approval timelines. The BLA was based on results from A-LONG, the largest registrational phase 3 clinical study in hemophilia A to date. In the A-LONG study, patients who injected Eloctate™ once-weekly to twice-weekly had low annualized bleeding rates. Prophylaxis in hemophilia A typically requires injections three times per week or every other day to maintain a sufficient circulating level of factor VIII, which prevents debilitating bleeding episodes.
• • On March 12, 2013, Biogen Idec has announced that the company has submitted a Biologics License Application (BLA) to the FDA for the marketing approval of recombinant factor VIII Fc fusion protein (rFVIIIFc) for the treatment of hemophilia A. Recombinant FVIIIFc is the first hemophilia A product candidate in a new class of long-lasting clotting factor therapies being developed with the goal of reducing the burden of treatment for this condition. The regulatory submission was based on results from A-LONG, the largest registrational phase 3 clinical study in hemophilia A to date. On March 4, 2013 Biogen Idec announced the FDA accepted for review the company’s BLA for its factor IX candidate, rFIXFc, for use in patients with hemophilia B.

Patents:

Submission of marketing authorization application USA : 2013-03-12

Submission of marketing authorization application UE: 2014-10-09

Withdrawal of marketing authorization application USA:

Withdrawal of marketing authorization application UE:

US authorization: 2014-06-06

UE authorization: 2015-11-19

Favourable opinion UE: 2015-09-24

Favourable opinion USA:

Orphan status USA: 2010-11-23

Orphan status UE: 2010-09-20

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes