Health Canada Approves Eloctate™, the First Therapy to Extend the Interval Between Prophylactic Infusions in Hemophilia A

– Only Hemophilia A Therapy to Provide Bleeding Protection with Prophylactic Regimens of Either Every Three to Five Days or Once-Weekly Infusions –

MISSISSAUGA, ON, Aug. 26, 2014 /CNW/ - Today Biogen Idec (NASDAQ: BIIB) announces that Health Canada has approved ELOCTATE™ [Antihemophilic Factor (Recombinant BDD), Fc Fusion Protein] for the control and prevention of bleeding episodes and routine prophylaxis in adults and children aged 12 and older with hemophilia A.¹  ELOCTATE is the first recombinant hemophilia A therapy with prolonged circulation in the body. It is the only treatment for hemophilia A to reduce the frequency of bleeding episodes via prophylactic regimens of either every three to five days, or once-weekly infusions. ELOCTATE offers people with hemophilia A the potential to extend the interval between prophylactic infusions.¹

"Prophylactic treatment is recommended for people with severe hemophilia; however, following a protective regimen can be a burden due to the frequency of infusions required," said Dr. Jean St-Louis, Director of the Hemostasis Programs of the CHU Sainte-Justine and the Maisonneuve-Rosemont Hospital. "Health Canada's approval of ELOCTATE provides people with hemophilia A an important new option in maintaining a prophylactic regimen, which may help alleviate this burden, and enable individuals to achieve their target factor levels with less frequent prophylactic dosing than traditional therapy."

In clinical trials, ELOCTATE was effective for both routine prophylaxis and to treat bleeding episodes with a favourable safety and tolerability profile. It was developed using a process called Fc fusion, which is designed to prolong the therapy's circulation in the body using a natural pathway.² The Health Canada approval of ELOCTATE follows regulatory approvals of Biogen Idec's hemophilia B therapy, ALPROLIX™ [Coagulation Factor IX (Recombinant), Fc Fusion Protein], in the United States, Canada, Japan and Australia.

"The Canadian Hemophilia Society (CHS) is very happy to see Health Canada's approval of ELOCTATE for the treatment of hemophilia A," said Craig Upshaw, CHS President. "This is the first in a promising new class of recombinant factor VIII products with extended half-life. It offers physicians and patients new options in care and the potential for more protective treatment regimens with less frequent infusions. We hope it will be available to Canadian patients in the very near future."

Hemophilia A is a rare, chronic, genetic disorder in which the ability of a person's blood to clot is impaired.³ This can lead to recurrent and extended bleeding episodes. Complications of bleeding episodes may range from severe swelling and pain to arthritis, joint damage, physical disability and death.^4,5,6,7 Therapies for hemophilia A can be administered either on a schedule to help prevent or reduce bleeding episodes (prophylaxis), or to control bleeding when it occurs (on-demand). The World Federation of Hemophilia recommends a prophylactic (prevention of bleeding) regimen as the goal of treatment for people with severe hemophilia.⁸

"Health Canada's approval of ELOCTATE marks the first significant hemophilia A treatment advance in more than 20 years," said Paul Petrelli, President and Managing Director, Biogen Idec Canada. "Biogen Idec is committed to improving the lives of people with severe chronic disorders, and we are excited to bring this important innovation and new treatment option for people living with hemophilia A to Canada."

Biogen Idec is committed to helping people with hemophilia A and is working through the required processes for review with Canadian Blood Services, the Service de Biovigilance du Ministère de la Santé et des Services sociaux and other provincial and national authorities to make ELOCTATE commercially available to all Canadians with hemophilia A.

About the A-LONG Study
The approval of ELOCTATE is based on results from the global, Phase 3 A-LONG clinical study, which included two Canadian sites. The A-LONG Study was an open-label, multi-centre study that examined the efficacy, safety and pharmacokinetics (PK) of ELOCTATE in 165 previously treated males aged 12 to 65 years with severe hemophilia A. PK is the measurement of the presence of the therapy in a person's body over time.

The study evaluated individualized and weekly prophylaxis to reduce or prevent bleeding episodes, and on-demand dosing to treat bleeding episodes. Results showed that adults and children (≥12 years) with severe hemophilia A achieved a statistically significant reduction of bleeding episodes in both of the study's prophylaxis arms: the median annualized bleeding rate (ABR) for individualized and weekly prophylaxis was 1.6 and 3.6, respectively; 45.3 per cent of patients on individualized and 17.4 per cent on weekly prophylaxis experienced no bleeding episodes. In addition, 98 per cent of bleeding episodes were controlled with one or two ELOCTATE infusions.¹

About ELOCTATE
ELOCTATE [Antihemophilic Factor (Recombinant BDD), Fc Fusion Protein] is the first recombinant clotting factor VIII therapy with prolonged circulation in the body. It is indicated for the control and prevention of bleeding episodes and routine prophylaxis in adults and children aged 12 and older with hemophilia A. ELOCTATE was developed by fusing B-domain deleted factor VIII to the Fc portion of immunoglobulin G subclass 1, or IgG₁ (a protein commonly found in the body). It is believed that this enables ELOCTATE to use a naturally occurring pathway to prolong the time the therapy remains in the body. While Fc fusion has been used for more than 15 years, Biogen Idec is the only company to apply it to the treatment of hemophilia.

Common adverse drug reactions observed in the clinical trial (≥1%) were arthralgia (joint pain) and malaise (general discomfort).

About Hemophilia A
The World Federation of Hemophilia global survey conducted in 2012 estimates that approximately 142,000 people are currently living with hemophilia A worldwide,⁹ including nearly 2,500 Canadians.¹⁰ It is caused by having substantially reduced or no factor VIII activity, which is needed for normal blood clotting. People with hemophilia A experience bleeding episodes that may cause pain, irreversible joint damage and life-threatening hemorrhages.⁴ Prophylactic infusions of factor VIII temporarily replace clotting factor necessary to control bleeding and prevent new bleeding episodes.^11,12

About Biogen Idec
Through cutting-edge science and medicine, Biogen Idec discovers, develops and delivers to patients worldwide innovative therapies for the treatment of neurodegenerative diseases, hemophilia and autoimmune disorders. Founded in 1978, Biogen Idec is the world's oldest independent biotechnology company. Patients worldwide benefit from its leading multiple sclerosis therapies. For product labeling, press releases and additional information about the company, please visit www.biogenidec.ca.

REFERENCES:

¹ ELOCTATE™ Product Monograph. August 22, 2014
² Mahlangu et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood Journal 2014 Nov; DOI 10.1182/blood-2013-10-529974.
³ Canadian Hemophilia Society. What is Hemophilia. Available at: http://www.hemophilia.ca/en/bleeding-disorders/hemophilia-a-and-b/what-is-hemophilia/. Accessed July 29, 2014.
⁴ Canadian Hemophilia Society. The Symptoms of Hemophilia. Available at: http://www.hemophilia.ca/en/bleeding-disorders/hemophilia-a-and-b/the-symptoms-of-hemophilia/. Accessed July 29, 2014.
⁵ Nilsson IM, Berntorp E, Löfqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe hemophilia A and B. J Intern Med 1992;232:25-32.
⁶ Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007;357:535-44.
⁷ Panicker J, Warrier I, Thomas R, Lusher JM. The overall effectiveness of prophylaxis in severe haemophilia. Haemophilia 2003;9:272-8.
⁸ About Bleeding Disorders – What is Prophylaxis. World Federation of Hemophilia. Available at: http://www.wfh.org/en/abd/prophylaxis/what-is-prophylaxis. Accessed July 30, 2014.
⁹ World Federation of Hemophilia. Annual Global Survey 2012. Available at: http://www1.wfh.org/publications/files/pdf-1574.pdf). Accessed July 29, 2014.
¹⁰ Canadian Hemophilia Registry – Hemophilia A. Available at: http://fhs.mcmaster.ca/chr/pdf/13/CHRVIII130517.pdf. Accessed July 29, 2014.
¹¹ Canadian Hemophilia Society. Factor Replacement Therapy. Available at: http://www.hemophilia.ca/en/bleeding-disorders/hemophilia-a-and-b/the-treatment-of-hemophilia/factor-replacement-therapy/#c236. Accessed July 29, 2014.
¹² Konkle et al. Hemophilia A. GeneReviews 2000. Available at: http://www.ncbi.nlm.nih.gov/pubmed/20301578. Accessed July 29.

SOURCE: Biogen Idec Canada

Canadian Media Contact: Rick Maddalena, [phone], [email protected]; French Canadian Media Contact: Joëlle Paquette, [phone], poste 33, [email protected]