INTRODUCTION Biogen Idec is the first biotechnology company to receive approval from the US Food and Drug Administration (FDA) for a long-acting recombinant coagulation factor IX product, that’s intended for use in patients with Hemophilia B (Christmas Disease). Approved under the proprietary name Alprolix, Coagulation Factor IX (Recombinant), Fc Fusion Protein is therapeutically designed to be prolonged in circulation and requires less recurrent injections by users. Alprolix is an innovation that is advancing treatments and the quality of life for patients with deficient or dysfunctional congenital Factor IX. SUBMISSION DETAILS Type of Submission BLA#BL STN 125444/0 Decision: Approved Date of Decision: 03/29/2014 Trade and Brand name: Active Ingredient: ALPROLIX™/Coagulation Factor IX (Recombinant), Fc Fusion Protein Recombinant fusion protein comprising the human Coagulation Factor IX sequence linked to the Fc domain of human immunoglobulin G1 (IgG1) Sponsor’s Name: Biogen Idec, Inc. Dosage form(s): Lyophilized powder with nominal potencies: 500 IU, 1000 IU, 2000 IU or 3000 IU per vial Approved Therapeutic use: Indicated in adults and children with Hemophilia B for: • Control and prevention of bleeding episodes, • Perioperative management, • Routine prophylaxis to prevent or reduce the frequency of bleeding episodes. ALPROLIX™ is not indicated for induction of immune tolerance in patients with Hemophilia B. Route(s) of administration: Intravenous Administration PDUFA Fee: PDUFA V PRODUCT BACKGROUND Alprolix is a sterile, lyophilized white powder for reconstitution with provided pre-filled diluents syringe, to render as a solution for an intravenous injection route of administration. It is manufactur... ... middle of paper ... ...Products/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM391049.pdf U.S. Food and Drug Administration. (2014, April 16). Summary Basis for Regulatory Action - ALPROLIX. Retrieved May 19, 2014, from ALPROLIX: http://www.fda.gov/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/ucm393432.htm US FDA. (2013, August 16). CLINICAL PHARMACOLOGY BLA REVIEW . (O. o. Research, Ed.) Retrieved May 19, 2014, from http://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM395349.pdf US. FDA. (2014, March 28). FDA approves first long-acting recombinant coagulation Factor IX concentrate for patients with Hemophilia B. Retrieved May 19, 2014, from http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm391037.htm
The cryoprecipitate is used for patients who don’t have enough or are unable to produce sufficient fibrinogen and clotting factor VIII. The FFP and cryoprecipitate can both be used for patients who have had a liver transplants and trauma cases in which the patients has clotting problems. The other products such as the immunoglobulin are used for fighting diseases because of the antibodies in the plasma. The anti-D is used in pregnancies in case of an RhD incompatibility between the baby and the mother; this stops the mother creating antibodies against the baby. The albumin and the clotting factors are used for patients who have a deficiency of these proteins.
was approved in 1982 and in another form, that is to be taken orally rather than
In some individuals with severe hemophilia, the factor VIII replacement therapy is identified as a foreign substance by their immune system. If this happens, their immune system will make antibodies against factor VIII. These antibodies will inhibit the ability of the factor to work in the clotting process. The higher the antibody or inhibitor level, the more factor VIII replacement therapy it takes to overcome the inhibition and produce clotting. This can complicate the treatment of a bleed. The good news is that there are different types of therapies available to successfully treat most individuals who develop inhibitors.
The U.S. Food and Drug Administration substantiated through a series of randomized clinical trials .
Hemophilia is a rare bleeding disorder that slows the blood clotting process, which is not normal. Some people with Hemophilia may just have a little bit of “clotting factor” or no clotting factor at all (National Institute of Health [NIH], 2013). Clotting factor is a protein in blood that controls bleeding and they are needed the blood to clot normally. In order to help the blood clot, clotting factors work with “platelets” (National Institute of Health [NIH], 2013). Platelets are small blood cell fragments that form in the bone marrow, a tissue in the bones that is similar to a sponge. The functions of platelets have a very important role in blood clotting; the role of a platelet is to stick together (by the help of clotting factors) to block cuts, break on the carriers of blood (veins or arteries) throughout the body, and stop the bleeding when “blood vessels” (National Institutes of Health [NIH], 2013) are injured. Blood vessels are tube like structures carrying blood through the tissues and organs, like a vein, artery, or capillary. People with hemophilia do not have enough “clotting factor VIII or IX” (World Federation of Hemophilia [WFH], 2013) in their blood, which results to prolonged bleeding or oozing, meaning that bleeding can last longer (though, not faster) than usual after surgeries, accidents, or having teeth pulled out at the dentist. Clotting factor VIII, which can also be called as “anti-hemophilic factor” (AHF, for short) (Patient.co.uk, 2011), is a blood clotting protein that is necessary for humans to have. Clotting Factor IX is a protein that i...
FDA, so many people were harmed. Even other countries versions of the FDA approved it which
Discuss the possible drug and excipient-related constrains of the formulation (no identity of the drug was given to you at this
...Through UDC,set of health monitoring data is collect from people with hemophilia and other bleeding disorders. The data collected is used to examine the severity of disease complications, describe treatment and care pattern , assesses quality of life and determine health issues for further future studies.( Centers for Disease Control and Prevention may,13,2013 1600 Clifton Rd. Atlanta, GA 30333, USA).
Turner, B. J., Newschaffer, C. J., Zhang, D., Fanning, T., & Hauck, W. W. (1999). Translating clinical trial results into practice. Annals of Internal Medicine, 130(12), 979-986.
review or pending approval unless the information has been in the public. The FDA has no legal
Clinical Trials and Human Subject Protection. (n.d.). Retrieved December 20, 2013, from Food and Drug Administration: http://www.fda.gov/ScienceResearch/SpecialTopics/RunningClinicalTrials/default.htm
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