Evaluation of Nonacog Beta Pegol Long-term Safety in the Immune-deficient Rowett Nude Rat (Crl:NIH- Foxn1rnu)

Toxicologic Pathology

Volumn 44, Issue 5, 2016, Pages 726-737

  Rasmussen, Caroline E ^a   Nowak, Jette ^a   Larsen, Julie M ^a   Bottomley, Anna ^b   Rowles, Alison ^b   Offenberg, Hanne ^a  

^a NOVO NORDISK A S   (Denmark)

^b HUNTINGDON LIFE SCIENCES   (United Kingdom)

Author keywords

athymic rat; Chronic toxicity; N9 GP; polyethylene glycol

Indexed keywords

RECOMBINANT BLOOD CLOTTING FACTOR 9; BLOOD CLOTTING FACTOR 9; MACROGOL DERIVATIVE; RECOMBINANT PROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CHOROID PLEXUS; CHRONIC TOXICITY; CONTROLLED STUDY; CYTOPLASM VESICLE; DRUG SAFETY; EPITHELIUM CELL; IMMUNOHISTOCHEMISTRY; MALE; NONHUMAN; PRIORITY JOURNAL; RAT; TRANSMISSION ELECTRON MICROSCOPY; ANIMAL; FEMALE; HUMAN; NUDE RAT;

ANIMALS; FACTOR IX; FEMALE; HUMANS; MALE; POLYETHYLENE GLYCOLS; RATS; RATS, NUDE; RECOMBINANT PROTEINS;

EID: 84975517850     PISSN: 01926233     EISSN: 15331601     Source Type: Journal    
DOI: 10.1177/0192623316633311     Document Type: Article

References (49)

1. Bartlett M. S., (1937). Properties of sufficiency and statistical tests. Proceedings of the Royal Society A: Mathematical, Physical and Engineering Sciences 160, 262-82.
2. Bendele A., Seely J., Richey C., Sennello G., Shopp G., (1998). Short communication: Renal tubular vacuolation in animals treated with polyethylene-glycol-conjugated proteins. Toxicol Sci 42, 152-57.
3. Berntorp E., Keeling D., Makris M., Tagliaferri A., Male C., Mauser-Bunschoten E. P., Musso R., Roca C. A., Hassoun A., Kollmer C., Charnigo R., Baumann J., Rendo P., (2012). A prospective registry of European haemophilia B patients receiving nonacog alfa, recombinant human factor IX, for usual use. Haemophilia 18, 503-9.
4. Bjoernsdottir I., Kappers W. A., Sternebring O., Kornoe H. T., Kristensen J. B., Thygesen P., Stow R., Fernyhough P., Bagger M., (2013). Excretion and pharmacokinetics of glycopegylated rFVIII (N8-GP) after single intravenous dose administration to rats. J Thromb Haemost 11, 579.
5. Center for Drug Evaluation and Research. (2007). BLA 125164 Pharmacology Review(s). Methoxypolyethylene Glycol Epoetin Beta (Mircera). Accessed August 13, 2015. http://www.accessdata.fda.gov/drugsatfda-docs/nda/2007/125164s000-pharmr-p1.pdf.
6. ® (peginesatide). Accessed August 13, 2015. http://www.accessdata.fda.gov/drugsatfda-docs/nda/2012/202799Orig1s000PharmR.pdf.
7. Chauhan A. N., Lewis P. D., (1979). A quantitative study of cell proliferation in ependyma and choroid plexus in the postnatal rat brain. Neuropathol Appl Neurobiol 5, 303-9.
8. Collins P. W., Young G., Knobe K., Karim F. A., Angchaisuksiri P., Banner C., Gursel T., Mahlangu J., Matsushita T., Mauser-Bunschoten E. P., Oldenburg J., Walsh C. E., Negrier C., (2014). Recombinant long-acting glycoPEGylated factor IX in hemophilia B: A multinational randomized phase 3 trial. Blood 124, 3880-86.
9. Dunnett C. W., (1955). A multiple comparison procedure for comparing several treatments with a control. J Am Stat Assoc 50, 1096-121.
10. Dunnett C. W., (1964). New tables for multiple comparisons with a control. Biometrics 20, 424-91.
11. European Medicines Agency. (2004a). EMEA 426716 EPAR Scientific Discussion. Neulasta (pegfilgrastim). Accessed August 13, 2015. http://www.ema.europa.eu/docs/en-GB/document-library/EPAR-Scientific-Discussion/human/000420/WC500025941.pdf.
12. European Medicines Agency. (2004b). EPAR Scientific Discussion. ReFacto (moroctocog alfa). Accessed August 13, 2015. http://www.ema.europa.eu/docs/en-GB/document-library/EPAR-Scientific-Discussion/human/000232/WC500049003.pdf.
13. European Medicines Agency. (2004c). EPAR Scientific Discussion. Somavert (pegvisomant). Accessed August 13, 2015. http://www.ema.europa.eu/docs/en-GB/document-library/EPAR-Scientific-Discussion/human/000409/WC500054625.pdf.
14. European Medicines Agency. (2005). EPAR Scientic Discussion. BeneFIX (Coagulation Factor IX [Recombinant]). Accessed August 13, 2015. http://www.ema.europa.eu/docs/en-GB/document-library/EPAR-Scientific-Discussion/human/000139/WC500020386.pdf.
15. European Medicines Agency. (2006). EMEA 671614 Scientific Discussion. Pegaptanib (Macugen). Accessed August 13, 2015. http://www.ema.europa.eu/docs/en-GB/document-library/EPAR-Scientific-Discussion/human/000620/WC500026218.pdf.
16. ® (certolizumab pegol). Accessed August 13, 2015. http://www.ema.europa.eu/docs/en-GB/document-library/EPAR-Public-assessment-report/human/001037/WC500069735.pdf.
17. European Medicines Agency. (2012). EMEA 647258 CHMP Safety Working Party's Response to the PDCO Regarding the Use of PEGylated Drug Products in the Paediatric Population. Accessed August 13, 2015. http://www.ema.europa.eu/docs/en-GB/document-library/Scientific-guideline/2012/11/WC500135123.pdf.
18. Fisher R. A., (1973). Statistical Methods for Research Workers. Hafner Publishing Company, New York, NY.
19. Furth R., (1989). Origin and turnover of monocytes and macrophages. In Cell Kinetics of the Inflammatory Reaction. (Iversen O. H.,), pp. 125-150. Springer-Verlag, Berlin and Heidelberg, Germany.
20. Hanes M. A., (2006). The nude rat. In The Laboratory Rat (Suckkow M. A., Weisbroth S. H., Franklin C. L., eds.), 2nd ed, pp. 733-59. Elsevier, Burlington, MA.
21. ICH (International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use). (2009). Guidance on Nonclinical Safety Studies for the Conduct of Human Clinical Trials and Marketing Authorization for Pharmaceuticals. Accessed August 13, 2015. http://www.ich.org/fileadmin/Public-Web-Site/ICH-Products/Guidelines/Multidisciplinary/M3-R2/Step4/M3-R2-Guideline.pdf.
22. ICH (International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use). (2011). ICH Harmonised Tripartite Guideline. Preclinical Safety Evaluation of Biotechnology-derived Pharmaceuticals S6 (R1). Accessed August 13, 2015. http://www.ich.org/fileadmin/Public-Web-Site/ICH-Products/Guidelines/Safety/S6-R1/Step4/S6-R1-Guideline.pdf.
23. Ivens I. A., Achanzar W., Baumann A., Brandli-Baiocco A., Cavagnaro J., Dempster M., Depelchin B. O., Rovira A. R., Dill-Morton L., Lane J. H., Reipert B. M., Salcedo T., Schweighardt B., Tsuruda L. S., Turecek P. L., Sims J., (2015). PEGylated biopharmaceuticals: Current experience and considerations for nonclinical development. Toxicol Pathol. August 3. pii: 0192623315591171. [. Epub ahead of print ].
24. Ivens I. A., Arora V., (2013). Blood products. In Nonclinical Development of Novel Biologics, Biosimilars, Vaccines and Specialty Biologics (Plitnick L. M., Herzyk D. J., eds.), pp. 287-301. Academic Press, San Diego, CA.
25. Jin K., Teng L., Shen Y., He K., Xu Z., Li G., (2010). Patient-derived human tumour tissue xenografts in immunodeficient mice: a systematic review. Clin Transl Oncol 12, 473-80.
26. Kirschbaum N., and U.S. Food and Drug Administration (2014). BLA 1125444 Summary Basis for Regulatory Action. ALPROLIX™/Coagulation Factor IX (Recombinant), Fc Fusion Protein. Accessed August 13, 2015. http://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM393434.pdf.
27. Latendresse J. R., Warbrittion A. R., Jonassen H., Creasy D. M., (2002). Fixation of testes and eyes using a modified Davidson's fluid: Comparison with Bouin's fluid and conventional Davidson's fluid. Toxicol Pathol 30, 524-33.
28. Lefkowitz J. B., Monroe D. M., Kasper C. K., Roberts H. R., (1993). Comparison of the behavior of normal factor IX and the factor IX Bm variant Hilo in the prothrombin time test using tissue factors from bovine, human, and rabbit sources. Am J Hematol 43, 177-82.
29. Liddelow S. A., (2015). Development of the choroid plexus and blood-CSF barrier. Front Neurosci 9, 32.
30. Monahan P. E., Liesner R., Sullivan S. T., Ramirez M. E., Kelly P., Roth D. A., (2010). Safety and efficacy of investigator-prescribed BeneFIX prophylaxis in children less than 6 years of age with severe haemophilia B. Haemophilia 16, 460-68.
31. Ostergaard H., Bjelke J. R., Hansen L., Petersen L. C., Pedersen A. A., Elm T., Moller F., Hermit M. B., Holm P. K., Krogh T. N., Petersen J. M., Ezban M., Sorensen B. B., Andersen M. D., Agerso H., Ahmadian H., Balling K. W., Christiansen M. L., Knobe K., Nichols T. C., Bjorn S. E., Tranholm M., (2011). Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide. Blood 118, 2333-41.
32. Osterud B., Kasper C. K., Lavine K. K., Prodanos C., Rapaport S. I., (1981). Purification and properties of an abnormal blood coagulation factor IX (factor IXBm)/kinetics of its inhibition of factor X activation by factor VII and bovine tissue factor. Thromb Haemost 45, 55-59.
33. Pilaro A. M., (2000). Toxicologists' Review. BLA 99-1488. Pegylated Interferon-α 2b (PEF-IFN, PEG-INTRON™). Accessed August 13, 2015. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/TherapeuticBiologicApplications/ucm094485.pdf.
34. ® for Hepatitis C. Accessed August 13, 2015. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/TherapeuticBiologicApplications/ucm094465.pdf.
35. Redzic Z. B., Segal M. B., (2004). The structure of the choroid plexus and the physiology of the choroid plexus epithelium. Adv Drug Deliv Rev 56, 1695-716.
36. Roth D. A., Kessler C. M., Pasi K. J., Rup B., Courter S. G., Tubridy K. L., (2001). Human recombinant factor IX: Safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates. Blood 98, 3600-06.
37. Rudmann D. G., Alston J. T., Hanson J. C., Heidel S., (2013). High molecular weight polyethylene glycol cellular distribution and PEG-associated cytoplasmic vacuolation is molecular weight dependent and does not require conjugation to proteins. Toxicol Pathol 41, 970-83.
38. Savient Pharmaceuticals I., (2009). KRYSTEXXA™ (pegloticase) for Intravenous Infusion. BLA No. 125293. Briefing Document for Arthritis Advisory Committee Division of Anesthesia, Analgesia, and Rheumatology Products FDA Arthritis Advisory Committee Meeting. Accessed August 13, 2015. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/ArthritisAdvisoryCommittee/UCM165814.pdf.
39. Schuurman H. J., (1995). The nude rat. Hum Exp Toxicol 14, 122-25.
40. Shirley E., (1977). A non-parametric equivalent of Williams' test for contrasting increasing dose levels of a treatment. Biometrics 33, 386-89.
41. Shultz L. D., Ishikawa F., Greiner D. L., (2007). Humanized mice in translational biomedical research. Nat Rev Immunol 7, 118-30.
42. Spector D. L., Goldman R. D., Leinwand L. A., (1998). Preparative methods for transmission electron microscopy. In Cells: A Laboratory Manual (Spector D. L., Goldman R. D., Leinwand L. A., eds.), Vol. 3, pp. 121.121-121.140. Cold Spring Harbor Laboratory Press, Cold Spring Harbor, NY.
43. Spitzer S. G., Warn-Cramer B. J., Kasper C. K., Bajaj S. P., (1990). Replacement of isoleucine-397 by threonine in the clotting proteinase factor IXa (Los Angeles and Long Beach variants) affects macromolecular catalysis but not L-tosylarginine methyl ester hydrolysis. Lack of correlation between the ox brain prothrombin time and the mutation site in the variant proteins. Biochem J 265, 219-25.
44. Steel R. G. D., (1959). A multiple comparison rank sum test: Treatments versus control. Biometrics 15, 560-72.
45. U.S. Food and Drug Administration (1997). BLA 96-1048 Summary Basis for Approval. Coagulation Factor IX (Recombinant) BeneFix™. Accessed August 13, 2015. http://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/ucm058970.pdf.
46. Webster R., Didier E., Harris P., Siegel N., Stadler J., Tilbury L., Smith D., (2007). PEGylated proteins: Evaluation of their safety in the absence of definitive metabolism studies. Drug Metab Dispos 35, 9-16.
47. Wilcoxon F., (1945). Individual comparisons by ranking methods. Biometrics Bull 1, 80-83.
48. Williams D. A., (1971). A test for differences between treatment means when several dose levels are compared with a zero dose control. Biometrics 27, 103-17.
49. Williams D. A., (1972). The comparison of several dose levels with a zero dose control. Biometrics 28, 519-31.