By: Benedette Cuffari, BS Toxicology Candidate c/o 2015
Hemophilia B is an inherited bleeding disorder that is caused by a substantially reduced or complete lack of blood clotting factor IX. Therefore, people suffering from hemophilia B experience bleeding episodes that cause pain, irreversible joint damage, and life threatening hemorrhages. Approximately 28,000 people are currently diagnosed with hemophilia B worldwide, most of them being male since the disease is inherited in an X-linked dominant way.1.
Hemophilia B is typically treated by periodically replacing the defective clotting factor IX with functional ones. These prophylactic infusions of factor IX are usually given once a week, but can be given up to two or three times a week depending on the severity of the patients’ bleeding episodes.1 Factor IX infusions can also be used to reduce bleeding levels as needed. On March 28, 2014, the FDA approved the release of AlprolixTM , or recombinant factor IX, as the first Hemophilia B treatment to greatly reduce the frequency of prophylaxis, as well as reduce the frequency of infusions needed for patients.2
The approval of AlprolixTM has been the first significant advance in hemophilia B treatment in over 17 years. The FDA states that “AlprolixTM is the first hemophilia B treatment designed to require less injections when used to prevent or reduce the frequency of bleeding”.2 This therapy is indicated for the control and prevention of bleeding episodes perioperative (surgical) management and routine prophylaxis in adults and children with hemophilia B.3
Alprolix TM was approved based on results from the global Phase 3 B-LONG study. The study results showed that adults and adolescents with severe hemophilia B were able to either prevent or substantially reduce their bleeding episodes with prophylactic infusions at least a week apart.3 A total of 123 individuals with severe hemophilia B were used as test subjects during the study, all of which experienced little to no side effects.3 after treatment of Alprolix TM. Some of the adverse reactions that were seen included headache, oral paresthesia (abnormal sensation in the mouth), dizziness, taste alteration, breath odor, fatigue, infusion site pain, palpitations, and hypotension. However, it is important to note that each event listed occurred in two or fewer study participants.
AlprolixTM is also approved in Canada, but is still under review in countries such as Australia and Japan. Researchers are very hopeful for the success of this drug, and the new relief that it will bring to hemophilia B patients around the world.
- National Hemophilia Foundation Staff. Hemophilia B. National Hemophilia Foundation. https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-B. Accessed January 4, 2014.
- FDA Staff. FDA approves first long-acting recombinant coagulation Factor IX concentrate for patients with Hemophilia B.U.S. Food and Drug Administration. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm391037.htm. Published March 28, 2014. Accessed January 4, 2014.
- Biogen Idec Staff. FDA Approves Biogen Idec’s Aliprolixä, The First Hemophilia B Therapy to Reduce Bleeding Episodes with Prophylactic Infusions Starting at Least a Week Apart. Biogen Idec. http://www.biogenidec.com/press_release_details.aspx?ID=14712&Action=1&NewsId=2303&M=NewsV2&PID=61997. Published March 28, 2014. Accessed January 4, 2014.
[pubmed_related keyword1=”recombinant” keyword2=”hemophilia” keyword3=”factor”]