The US Food and Drug Administration (FDA) has accepted the Biogen Idec's Biologics License Application (BLA) for the marketing approval of Eloctate (recombinant factor VIII Fc fusion protein) for the treatment of haemophilia A.
Eloctate is the first haemophilia A product candidate in a new class of long-lasting clotting factor therapies being developed with the goal of providing long-lasting protection and reducing the burden of treatment for patients with this chronic condition.
“Eloctate has the potential to improve adherence by reducing the number of intravenous injections needed to prevent bleeds, which is an important need for people with haemophilia A,” said Glenn Pierce, senior vice president of global medical affairs and chief medical officer of Biogen Idec’s haemophilia therapeutic area. “For those people currently on preventative—or prophylactic—treatment, Eloctate provides the potential to reduce the number of intravenous injections by 50 to 100 per year.”
The Eloctate BLA was based on results from A-LONG, the largest registrational phase 3 clinical study in haemophilia A to date. In the A-LONG study, patients who injected Eloctate once-weekly to twice-weekly had low annualized bleeding rates. Prophylaxis in haemophilia 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.
With the FDA’s acceptance of the Eloctate BLA, Biogen Idec now has product candidates for both haemophilia A and B under review with the agency. On March 4, 2013, the company announced that the FDA accepted for review the BLA for its factor IX candidate, Alprolix (recombinant factor IX Fc fusion protein), for the treatment of haemophilia B. Both applications were granted standard review.
Eloctate is a clotting factor developed using Biogen Idec’s novel and proprietary monomeric Fc fusion technology, which makes use of a naturally occurring pathway to delay the destruction of factor and cycles it back into the bloodstream, resulting in a longer circulating half-life.
With this technology, Eloctate is designed to provide long-lasting protection from bleeding and reduce the treatment burden associated with haemophilia A, which currently can require approximately 150 to 180 injections annually for prophylaxis with commercially available factor VIII products. Fc fusion technology is used in seven FDA-approved products for the long-term treatment of chronic diseases including rheumatoid arthritis, psoriasis and platelet disorders.
Haemophilia A is a rare, inherited 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 is caused by having substantially reduced or no factor VIII activity, which is needed for normal blood clotting. People with haemophilia A therefore need injections of factor VIII to restore the coagulation process and prevent frequent bleeds that could otherwise lead to pain, irreversible joint damage and life-threatening haemorrhages. The Medical and Scientific Advisory Council of the National Haemophilia Foundation recommends prophylaxis as the optimal therapy for people with severe haemophilia A.
Biogen Idec and Swedish Orphan Biovitrum (Sobi) are partners in the development and commercialization of Alprolix in haemophilia B and Eloctate in haemophilia 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 the right to opt in to assume final development and commercialization in Europe including Russia, the Middle East and Northern Africa.
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