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
...-1 (PAI-1) from the endothelial cells and monocytes, activating the extrinsic coagulation pathway. This also leads to activation of factor X and fibrin production.
Furosemide is available in tablets, sublingual tablets, oral suspension, and intravenously. (2) The recommended schedule dose for adults for an initial dose is 20 to 80mg. (3) The same dose as well as higher doses may be administered 6 to 8 hours following the previous dose if needed. (2) When titrating doses it may be raised by 20 to 40mg but not within 6 to 8 hours after the initial dose, and this may continue until the desi...
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.
U.S. Food and Drug Administration. “CFR -- Code of Federal Regulations Title 21” (21CFR101.9). U.S. Food and Drug Administration. U.S. Department of Health & Human Services, 1 Apr. 2013. Web. 21 Mar. 2014.
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 .
review or pending approval unless the information has been in the public. The FDA has no legal
FDA, so many people were harmed. Even other countries versions of the FDA approved it which
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
The changes in f8 and f9 gene are responsible for hemophilia A and B. the f8 gene codes for a protein called coagulation 8. This protein is responsible for the blood clotting process. After an injury blood clots protect the body by sealing off the area of injury. Mutation in the f8 and f9 gene can lead to the abnormal form of the protein. Also can lead to a reduction of the coagulation pro...
The Merck Manuals. (2008). The Merck Manuals. Retrieved from The Merck Manuals Online Medical Library: http://www.merckmanuals.com/home/ag/sec07/ch107/ch107b.html
U.S. National Library of Medicine, 26 Sept. 2011. Web. The Web. The Web. 19 Nov. 2013.
Mark H. Beers, MD. "The Merck Manual of Medical Information." Mark H. Beers, MD. The Merck Manual of Medical Information. Whitehouse Station NJ: Merck & CO., INC, 2003. 1630-1631.
Clinical trials containing new drugs are categorized into essential four phases (1). The drug development procedures are usually performed through the all phases over several years (1). The drug will be approved for use in the general population, if it successfully exceeds through the first three phases; I, II, and III. The fourth phase -IV- is called Post marketing Surveillance and/or Post approval studies (2). Postmarketing Surveillance Trial provides the safety surveillance (Pharmacovigilance) (3). phase IV Analytics methods studies may be required by sponsoring companies, when detecting a new drug market or testing the drug interactions with other drugs, and also by regulatory authorities (4). The safety surveillance, which is a very important part of a comprehensive post-marketing program, investigates drugs long term effects in a large number of people (1) (3).
"MedlinePlus - Health Information from the National Library of Medicine." National Library of Medicine - National Institutes of Health. Web. 06 Mar. 2011.