Hemophilia is a type of bleeding disorder that causes the blood to take a long time to clot, and occurs almost exclusively in males. People with hemophilia B have a deficiency in clotting factor IX, a specific protein in the blood.1 According to the World Federation of Hemophilia, approximately one in 50,000 people globally2 have hemophilia B, including nearly 4,110 people living with hemophilia B in the U.S.3
People with hemophilia face specific risks and need to be careful not to cause injury to their bodies, as injuries can prompt a bleed. Bleeding in hemophilia can be potentially life-threatening. While people with hemophilia do not bleed faster than people without hemophilia, they do tend to bleed longer because of their inability to clot, increasing their risk of internal injury.1
In the last century alone, the treatment of hemophilia has undergone many changes. In the 1950s, plasma was first used to treat people with hemophilia. 4 In the 1960s, researchers began experimenting with concentrates of factor, 4 and in the early 1990s, the first recombinant factor products became available. 4 Innovations continue to be explored in the manufacturing and purification processes of hemophilia treatments.
A documentary, titled “Pushing Boundaries: How Science Impacted the Treatment of Hemophilia B” presents a brief history of hemophilia B treatment and tells the story of the development of recombinant factor IX and the approval of BeneFIX® Coagulation Factor IX (Recombinant). BeneFIX is a recombinant coagulation factor IX product indicated for the control, prevention and perioperative management of bleeding episodes in adult and pediatric patients with hemophilia B. BeneFIX received FDA fast track approval in the U.S. on February 11, 1997. This year, BeneFIX celebrates its 15th anniversary.
The documentary also features interviews with researchers and hemophilia B patients, while illustrating the innovative science brought forth by researchers in developing BeneFIX.
BeneFIX was developed by Genetics Institute (now Pfizer) in Andover, Massachusetts, at a time when few people had ever manufactured and formulated a recombinant protein clotting factor. The development of BeneFIX pioneered many of the recombinant protein drug production techniques commonly used today. BeneFIX is currently marketed in 34 countries and has been used by more than 30,000 patients worldwide.5
Hemophilia B patient Phil H. began using BeneFIX following its approval in 1997. Patients like Phil and other members of the hemophilia B community have provided feedback over the years that has not only contributed to the development of BeneFIX, but also to its latest product offerings. These offerings include the development of the Rapid Reconstitution (R2) kit, the ability to store at room temperature, and the 3000 IU dosage strength.
“Growing up, it was challenging to deal with my bleeds,” said Phil. “I’m thankful that my voice and the voice of patients like me was heard.”
BeneFIX is an injectable medicine that is used to help control and prevent bleeding in people with hemophilia B. Hemophilia B is also called congenital factor IX deficiency or Christmas disease.
BeneFIX is NOT used to treat hemophilia A.
Important Safety Information for BeneFIX
- BeneFIX is contraindicated in patients who have manifested life-threatening, immediate hypersensitivity reactions, including anaphylaxis, to the product or its components, including hamster protein.
- Call your health care provider right away if your bleeding is not controlled after using BeneFIX.
- Allergic reactions may occur with BeneFIX. Call your health care provider or get emergency treatment right away if you have any of the following symptoms: wheezing, difficulty breathing, chest tightness, your lips and gums turning blue, fast heartbeat, facial swelling, faintness, rash or hives.
- Your body can make antibodies, called “inhibitors,” which may interfere with the effectiveness of BeneFIX.
- If you have risk factors for developing blood clots, such as a venous catheter through which BeneFIX is given by continuous infusion, BeneFIX may increase the risk of abnormal blood clots. The safety and efficacy of BeneFIX administration by continuous infusion have not been established.
- Some common side effects of BeneFIX are nausea, injection site reaction, injection site pain, headache, dizziness and rash.
Please see full Prescribing Information for BeneFIX available at www.benefix.com.
1 National Heart Lung and Blood Institute. “What is Hemophilia.” Accessed 5 March 2012. Available at http://www.nhlbi.nih.gov/health/health-topics/topics/hemophilia/
2 World Federation of Hemophilia. “Frequently Asked Questions.” Accessed 28 June 2012. Available at http://www.wfh.org/en/page.aspx?pid=637
3 Centers for Disease Control and Prevention. “Summary Report of UDC Activity, National Patient Demographics (Hemophilia).” Accessed 11 July 2012. Available at https://www2a.cdc.gov/ncbddd/htcweb/UDC_Report/UDC_Report.asp
4 National Hemophilia Foundation. “History of Bleeding Disorders.” Accessed 5 August 2012. Available at http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=178&contentid=6
5Periodic Safety Update Report #19. Worldwide Marketing Authorization Status.