Today, Biogen Idec announced that its Biologics License Application (BLA) has been accepted by the US Food and Drug Administration (FDA) for the marketing approval of its hemophilia A treatment.

Eloctate (recombinant factor VIII F c fusion protein) is the first hemophilia A product candidate in a new class of long-lasting clotting factor therapies being developed to provide long-lasting production and reduce the burden of treatment for hemophilia A patients. The drug candidate uses a naturally occurring pathway to delay destruction of factor and cycles it back into the bloodstream for a longer circulating half-life.

“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 hemophilia A,” Glenn Pierce, MD, PhD, Senior Vice President of Global Medical Affairs and Chief Medical Officer at Biogen Idec’s hemophilia therapeutic area stated. “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.”

Current hemophilia A treatments can require approximately 150 to 180 injections per year for prophylaxis factor VIII products.

The acceptance of the Eloctate’s BLA was based on results from a Phase III clinical study known as A-LONG. This is the largest registrational Phase III clinical study in hemophilia A. Results found that patients who injected the drug once-weekly to twice-weekly had low annualized bleeding rates. Hemophilia A patients typically require prophylaxis injections three times per week or every other day to keep a sufficient circulating level of factor VIII.

Factor VIII is needed to prevent bleeding episodes for patients with this rare, inherited disorder that impairs a person’s blood to clot. Hemophilia A occurs in roughly one in 5,000 male births per year. If untreated, bleeding events could lead to pain, irreversible joint damage, and life-threatening hemorrhages.

In addition to Eloctate, Biogen’s hemophilia B candidate’s BLA was recently accepted by the FDA. Alprolix’s (recombinant factor IX Fc fusion protein) BLA was accepted by the FDA on March 4. Both products are being developed and commercialized in partnership by Biogen Idec and Swedish Orphan Biovitrum (Sobi). Biogen leads development, has manufacturing rights and commercial rights in North America and all other regions excluding the Sobi territory.

Source: Biogen Idec

Last Updated: 5/13/13; 2:40PM EST

AMA Enterprise, a division of the American Management Association, has partnered with Biogen Idec to develop a series of workshops for patients with bleeding disorders.

The programs are designed to provide practical life skills to enable hemophilia patients to live life on their own terms. The development of the four-workshop program is based on input from members of the hemophilia community. Lessons from respected authors, universities and community and business leaders addressing core life skills are incorporated into the workshops.

Workshops focus on several topics, such as how to make a great impression in numerous situations, how to express oneself and appropriately manage emotions during common interactions and challenging situations, how to create win-win conversations to resolve conflicts and be more persuasive, and how to help individuals identify inner strength and learn to assert them.

“We are honored to have been chosen by Biogen Idec to provide educational services to this community. Being able to teach empowering life skills to individuals who live with – but refuse to be limited by – a chronic condition is part of our expanded focus at AMA and one we take particular pride in,” Cheryl Stern, Regional Director at AMA Enterprise said in a statement. “These programs provide valuable tools to empower individuals to build confidence and life skills to live more empowered lives and advocate for themselves and their families.”

According to Jess Swann, Director of Marketing & Community Relations at Biogen Idec, programs are based on input from those living with bleeding disorders and their loved ones in order to meet specific needs within the community.

“For Biogen Idec, these workshops are aligned with our mission to empower the approximately 20,000 people with hemophilia throughout the United States to live the lives they choose. This is an area we’re deeply committed to,” Swann stated.

According to Stern, there have already been three pilot training workshops delivered with over 120 participants.

“Early feedback demonstrates that these workshops have a positive impact. By an overwhelming margin, participants found the content to be relevant (93%), the facilitation effective (94%). Overall, satisfaction was very high (96%), and participants felt that the skills introduced helped them think and behave differently in addressing life, family and work issues,” Stern said.

According to AMA, workshops will be conducted throughout 2013 at various locations in the US. Biogen has a number of programs for people living with inherited bleeding disorders in addition to the life skills workshop.

Source: American Management Association

Last Updated: 4/17/13; 11:15AM EST

On World Hemophilia Day 2013, we recognize the World Federation of Hemophilia (WFH) and the global community who have worked together for the last 50 years to improve care and treatment for inherited bleeding disorders.

This year, April 17^th takes on a special significance as we mark ’50 Years of Advancing Treatment for All’. Sponsors of this year’s World Hemophilia Day include Baxter, Bayer, Biogen Idec, CSL Behring, Novo Nordisk, Pfizer, Precision BioLogic, and Sobi.

The WFH launched the hemophilia awareness video, The Journey Begins, to honor its 50^th anniversary. The video explores the origins of the WFH and how Frank Schnabel brought together the global bleeding disorders community to form WFH. World Hemophilia Day raises awareness about where the hemophilia community has been, and where WFH wants to go. By coming together, the community can close the gap in care for hemophilia patients.

Significant progress has been made in hemophilia treatment over the past 50 years; however the vast majority of people living in developing countries with inherited bleeding disorders do not have access to the proper care. Approximately 75 percent of people worldwide, with a bleeding disorder, still receive inadequate treatment, or no treatment at all.

“One of the greatest benefits of participating in World Hemophilia Day is the recognition that our community is more powerful when we work together,” Alain Weill, President of WFH said in a news release. “People from around the world will mark this day and raise awareness about the need to achieve Treatment for All.”

WFH is offering individuals in the US memberships for $50, in honor of its 50^th anniversary. Membership benefits include WFH’s newsletter Hemophilia World, The WFH Annual Report, WFH e-newsletters and e-bulletins, discounts on subscriptions to WFH’s official journal Haemophilia, discounts on WFH publications, and discounts on registration fees for WFH events. To support WFH and assist in the goal to “Close the Gap,” click here.

Source: World Federation of Hemophilia (WFH)

Last Updated: 4/17/13; 10:15AM EST

Biogen Idec recently announced that the company has submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for its hemophilia drug.

The BLA is 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 this new class of long-lasting clotting factor therapies. These new therapies are being developed with a goal of reducing the burden of treatment for this condition.

If the FDA approves the product, Biogen’s rFVIIIFc will be the first major advance in treatment for hemophilia A patients in over twenty years. The company said that Biogen submitted the BLA based on results from A-LONG, which is the largest registrational phase III study in hemophilia A to date.

“This regulatory submission marks another significant step toward our goal of transforming the care of hemophilia for patients, families and caregivers,” Glenn Pierce, MD, PhD, senior vice president of Global Medical Affairs and chief medical officer of Biogen Idec’s hemophilia therapeutic area stated. “In our phase 3 study, patients treated with rFVIIIFc were able to inject rFVIIIFc once-weekly to twice-weekly, which creates the potential for those currently on prophylactic treatment to reduce injections by 50 to 100 per year. Moreover, patients currently treating bleeding episodes could potentially dose prophylactically once per week and maintain significant protection from bleeding with about the same total number of injections each year they use to treat bleeding episodes today.”

Typically, patients with hemophilia A require prophylaxis injections three times per week or every other day to maintain a sufficient circulating level of clotting factor to protect them from bleeding. Without prophylactic treatment, these patients are at risk of bleeding episodes that can cause major damage, such as irreversible joint damage and life threatening hemorrhages.

Additionally, the company announced on March 4, 2013 that the FDA accepted its BLA for review of its factor IX candidate, rFIXFc, for use in patients with hemophilia B.

Hemophilia A is a rare, inherited disorder which occurs in roughly one in 5,000 male births annually due to substantially reduced or no factor VIII activity that is needed for normal blood clotting.

Source: Biogen Idec

Last Updated: 3/13/13; 11:40AM EST