Swedish Orphan Biovitrum Ltd (Sobi™), Cambridge UK, today announce that Elocta® (efmoroctocog alfa), the first recombinant human factor VIII Fc-fusion protein with an extended half-life for the treatment of haemophilia A, and Alprolix® (efrenonocog alfa), recombinant human factor IX Fc-fusion protein with extended half-life for haemophilia B, have been approved for reimbursement by the NHS across the UK. NHS England has now confirmed reimbursement alongside Scotland, Wales and Northern Ireland where Elocta and Alprolix were already available.
This expansion in availability in the UK 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). Since first becoming commercially available, more than 2,700 patients have now been treated in a real-world setting with Elocta, corresponding to about 1,800 patient-years of experience. In addition more than 1,000 patients already have real world experience with Alprolix.
“We are really pleased to see the UK join the growing list of countries with access to extended half- life factors for people living with Haemophilia”, said Liz Carroll, Chief Executive of The Haemophilia Society. “It is important that Haemophilia treatment and care continues to move forward and new developments in treatment are accessible within the NHS as a whole. We will be publishing factsheets for members, explaining these new therapeutic options and stand ready to support members and their families as they consider these new treatments”
Dr Gary Benson, Haemophilia Centre Director Royal Belfast City Hospital said: “Extended half-life factors could give us a new way to improve care and outcomes for people with haemophilia, using an individualised approach at no extra cost to the NHS. We can significantly reduce the number of intravenous infusions needed, whilst also in many patients aim for increased protection from debilitating bleeding events.”
“Making these new treatments available on the NHS in Scotland is a big step forward in improving the lives of people living with haemophilia,” said Dan Farthing-Sykes, Chief Executive of Haemophilia Scotland. “They offer the prospect of fewer injections and the potential for better protection from painful internal bleeding. We know families all over Scotland are very keen to find out if they could benefit from them. The fact they are now available to Scottish Haemophilia Centres means Scotland’s fantastic specialist teams will be able to work with patients to understand how they can be used most effectively.”
“This extensive post approval clinical experience with Elocta and Alprolix complements the clinical data generated by our pivotal clinical studies, and follows the findings of the long term ASPIRE and B- YOND extension studies. We believe that this comprehensive clinical data and real world experience can provide support to clinicians and patients while making their treatment choices,” said Krassimir Mitchev, MD, PhD, vice president and medical therapeutic area head of Haemophilia at Sobi.
Through the pivotal clinical trials (A-LONG, Kids A-LONG, B-LONG, Kids B-LONG) and extension studies (ASPIRE and B-YOND) 233 patients have been treated with Elocta and 153 with Alprolix in a clinical trial setting.
Eloctate was first approved for haemophilia A in the USA in June 2014 and in the EU (as Elocta) in November 2015. Alprolix was first approved for the treatment of Haemophilia B in the USA in March 2014 and in the EU in May 2016.
About Haemophilia A & B
Haemophilia is a rare, genetic disorder in which the ability of a person's blood to clot is impaired. Haemophilia A occurs in about one in 5,000 male births annually, and more rarely in females. Haemophilia B occurs in about one in 25,000 make births and more rarely in Females.
People with haemophilia A and B experience prolonged bleeding episodes that can cause pain, irreversible joint damage and life-threatening haemorrhages. Prophylactic infusions of factor VIII or IX can temporarily replace the missing clotting factors that are needed to control bleeding and prevent new bleeding episodes.i The World Federation of Hemophilia recommends prophylaxis as the optimal therapy as it can prevent bleedings and joint destruction.ii
Elocta (efmoroctocog alfa), the first recombinant clotting factor VIII therapy that offers an extended half-life in the body, is approved in the European Union, Switzerland, Iceland, Liechtenstein and Norway, as well as the United States, Canada, Australia, New Zealand and Japan (as Eloctate). It was developed for haemophilia A by fusing factor VIII to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body). This enables Elocta to use a naturally occurring pathway to prolong the time the therapy remains in the body. While Fc fusion technology has been used for more than 15 years, Sobi and Biogen are the first companies to utilise it in the treatment of haemophilia.
As with any factor replacement therapy, development of inhibitors may occur following administration of Elocta/Eloctate
Alprolix® (eftrenonacog alfa) is a recombinant clotting factor therapy developed for haemophilia B currently approved for the treatment of haemophilia B in the European Union as well as Iceland, Liechtenstein and Norway, as well as the US, Canada, Japan, Australia, New Zealand. It was developed by fusing factor IX to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body). This enables Alprolix to use a naturally occurring pathway to prolong the time the therapy remains in the body. While Fc fusion technology has been used for more than 15 years, Sobi and Biogen are the first companies to utilise it in the treatment of haemophilia.
As with any factor replacement therapy, allergic-type hypersensitivity reactions and development of inhibitors may occur following administration of Alprolix
i World Federation of Hemophilia. About Bleeding Disorders – Frequently Asked Questions. Available at: http://www.wfh.org/en/page.aspx?pid=637#Difference_A_B. Accessed on: June 17, 2016.
ii Guideline for the management of hemophilia, World Federation of Hemophilia, 2nd edition, http://www1.wfh.org/publication/files/pdf-1472.pdf. Besöktes i december 2015