BLOOD PRODUCTS ADVISORY COMMITTEE
January 9, 2009
Topic I: CSL Behring’s Biologics License Application for Plasma-Derived Fibrinogen
Concentrate for Treatment of Bleeding in Congenital Fibrinogen
1. Acharya SS, Coughlin A, Dimichele DM, North American Rare Bleeding Disorder Study Group. Rare Bleeding Disorder Registry: deficiencies of factors II, V, VII, X, XIII, fibrinogen and dysfibrinogenemias. Journal of Thrombosis and Haemostasis 2004; 2: 248-56.
2. Carroll RC, Craft RM, Chavez JJ, et al. Measurement of functional fibrinogen levels using the Thrombelastograph. Journal of Clinical Anesthesia 2008; 20: 186-90.
3. Fleming TR. Surrogate Endpoints and FDA’s Accelerated Approval Process: The Challenges are greater than they seem. Health Affairs 2005: 24, no.1: 67-78.
4. Kalina U, Stöhr HA, Bickhard H, et al. Rotational thromboelastography for monitoring of fibrinogen concentrate therapy in fibrinogen deficiency. Blood Coagulation and Fibrinolysis 2008; 9:777-83.
5. Kreuz W, Meili E, Peter-Salonen K, et al. Efficacy and tolerability of a pasteurised human fibrinogen concentrate in patients with congenital fibrinogen deficiency. Transfusion and Apheresis Science 2005; 32: 247-53.
6. Kreuz W, Meili E, Peter-Salonen K, et al. Pharmacokinetic properties of a pasteurised fibrinogen concentrate. Transfusion and Apheresis Science 2005; 32: 239-46.
Topic II: GTC Biotherapeutics Inc. Biologics License Application for Recombinant Antithrombin III Derived from Genetically Engineered Goats for Treatment of Patients with Hereditary Antithrombin III Deficiency to Prevent Thrombosis During High Risk Situations like Surgery and Obstetrical Procedures
1. Dingermann, T. Recombinant therapeutic proteins: Production platforms and challenges. Biotechnology Journal 2008; 3: 90-97.
2. Food and Drug Administration, Center for Veterinary Medicine. Draft Guidance for Industry: Regulation of Genetically Engineered Animals Containing Heritable rDNA Constructs. Issued, September 18, 2008. (http://www.fda.gov/cvm/Guidance/guide187.pdf or html )
3. Houdebine, LM. Production of pharmaceutical proteins by transgenic animals. Comparative Immunology Microbiology and Infectious Disease 2008; doi:10.1016/j.cimid.2007.11.005 (in press.)
5. Patnaik MM, Moll S. Inherited antithrombin deficiency: a review. Haemophilia 2008; 14: 1229–1239.