Regulatory Forum Opinion Piece*:Differences between Protein-based Biologic Products (Biotherapeutics) and Chemical Entities (Small Molecules) of Relevance to the Toxicologic Pathologist

Toxicologic Pathology

Volumn 41, Issue 1, 2013, Pages 128-136

  Leach, Michael W ^a  

^a Drug Safety Research and Development   (United States)

Author keywords

biotechnology; development; drug development; immunopathology; preclinical research; toxicologic pathology

Indexed keywords

ALEMTUZUMAB; ANTIGEN; DRUG; ERYTHROPOIETIN; IMMUNOGLOBULIN E; IMMUNOGLOBULIN G; IMMUNOMODULATING AGENT; MACROGOL; MONOCLONAL ANTIBODY; NUCLEIC ACID; PROTEIN; PROTEIN BASED BIOLOGIC AGENT; RECOMBINANT DNA; SUGAR; UNCLASSIFIED DRUG; VACCINE;

ALLERGIC REACTION; ANAPHYLAXIS; APLASTIC ANEMIA; ARTICLE; BACTERIUM; CELL CULTURE; CYTOMEGALOVIRUS; DEVELOPMENTAL TOXICITY; DOSE RESPONSE; DRUG DETERMINATION; DRUG EFFECT; DRUG HALF LIFE; DRUG INDUSTRY; DRUG METABOLISM; DRUG STRUCTURE; DRUG SYNTHESIS; ENVIRONMENTAL FACTOR; EXPERIMENTAL DOG; EXPERIMENTAL MONKEY; HUMAN; IMMUNE RESPONSE; IMMUNOGENICITY; IMMUNOPATHOLOGY; IMMUNOPRECIPITATION; IMMUNOSUPPRESSIVE TREATMENT; IN VIVO STUDY; INSECT; LEUKOCYTE; MAMMAL CELL; METHODOLOGY; MOLECULAR LIBRARY; MORTALITY; NONHUMAN; PATHOLOGIST; PLANT; PLASMODIUM; PRIORITY JOURNAL; PROTEOMICS; REPRODUCTIVE TOXICITY; VASCULITIS; YEAST;

ANIMALS; BIOLOGICAL ASSAY; BIOMEDICAL RESEARCH; BIOSIMILAR PHARMACEUTICALS; BIOTECHNOLOGY; DOSE-RESPONSE RELATIONSHIP, DRUG; DRUG DISCOVERY; HUMANS; TOXICOLOGY;

EID: 84871661130     PISSN: 01926233     EISSN: 15331601     Source Type: Journal    
DOI: 10.1177/0192623312451371     Document Type: Article

References (28)

1. Bell G. M. Reynolds G. Isaacs J. D. (2011). Biologic therapies in non-rheumatic diseases: Lessons for rheumatologists? Nature Rev Rheumatol 7, 507–16.
2. Bugelski P. J. Treacy G. (2004). Predictive power of preclinical studies in animals for the immunogenicity of recombinant therapeutic proteins in humans. Curr Opin Mol Ther 6, 10–16.
3. Bussiere J. L. Leach M. W. Price K. D. Mounho B. J. Lightfoot-Dunn R. (2011). Survey results on the use of the tissue cross-reactivity immunohistochemistry assay. Reg Toxicol Pharmacol 59, 493–502.
4. Cannon K. (2012). Neurologic issues associated with biotherapeutics. Paper presented at the Spring 2012 Boston Area Pharmaceutical Toxicology Group Meeting, March 23, 2012, Cambridge, MA, http://docs.google.com/file/d/0B_2H4MkbZD-EdmRmeTlmd20zVmM/edit?pli=1.
5. Cavagnaro J. (2010). Considerations for the preclinical safety evaluation of biopharmaceuticals. In Comprehensive toxicology. Toxicity testing and evaluation (Lamb J., ed., McQueen C. A., editor in chief), 2nd ed., Vol. 3, pp. 29–52. Elsevier Science, Kidlington, United Kingdom.
6. Clarke J. B. (2010).Mechanisms of adverse drug reactions to biologics. Handb Exp Pharmacol 196, 453–74.
7. Demeule B. Gurny R. Arvinte T. (2006). Where disease pathogenesis meets protein formulation: Renal deposition of immunoglobulin aggregates. Eur J Pharm Biopharm 62, 121–30.
8. EMA (2010). Draft guideline on similar biological medicinal products containing monoclonal antibodies (EMA/CHMP/BMWP/403543/2010).
9. EMEA (2007). Guideline on strategies to identify and mitigate risks for first-in-human clinical trials with investigational medicinal products, http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500002988.pdf.
10. EMEA (2009). Assessment Report for Cimzia, EMEA Doc.Ref.: EMEA/664021/2009, http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/001037/WC500069735.pdf.
11. FDA (2011). Brentuximab vedotin pharmacology review(s) Application Number: 125388Orig1s000, accessed January 31, 2012, http://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/125388Orig1s000PharmR.pdf.
12. FDA (2012a). Drugs@FDA Glossary of Terms, accessed January 30, 2012, http://www.fda.gov/Drugs/informationondrugs/ucm079436.htm.
13. FDA (2012b). Draft guidance for industry. Scientific considerations in demonstrating biosimilarity to a reference product.
14. Hu X. Olivier K. Polack E. Crossman M. Zokowski K. Gronke R. S. Parker S. Li Z. Nestorov I. Baker D. P. Clarke J. Subramanyam M. (2011). In vivo pharmacology and toxicology evaluation of polyethylene glycol-conjugated interferon beta-1a. J Pharmacol Exp Ther 338, 984–96.
15. International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH). S6 Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals. (1997), http://www.fda.gov/downloads/regulatoryinformation/guidances/ucm129171.pdf.
16. International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH). S6(R1) Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals. (2011), http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Safety/S6_R1/Step4/S6_R1_Guideline.pdf.
17. Kumar V. Abbas A. K. Fausto N. Aster J. C. (2010). Robbins and Cotran pathologic basis of disease, professional edition, 8th ed. Chapter 6, 183–257—Diseases of the immune system (Kumar V. Abbas A. K. Fausto N. Aster J. C., eds). Copyright 2010 by Saunders. Philadelphia, PA.
18. Leach M. W. (2010). Immunogenicity-related effects in the liver of rats administered a human monoclonal antibody with a bell-shaped dose-response pattern. (2010). Paper presented at BioSafe General Membership Meeting, Cambridge, Massachusetts, April 28–30.
19. Leach M. W. Halpern W. G. Johnson C. W. Rojko J. L. MacLachlan T. K. Chan C. M. Galbreath E. J. Ndifor A. M. Blanset D. L. Polack E. Cavagnaro J. A. (2010). Use of tissue cross-reactivity studies in the development of antibody-based biopharmaceuticals: History, experience, methodology, and future directions. Toxicol Pathol 38, 1138–66.
20. Ponce R. Abad L. Amaravadi L. Gelzleichter T. Gore E. Green J. Gupta S. Herzyk D. Hurst C. Ivens I. A. Kawabata T. Maier C. Mounho B. Rup B. Shankar G. Smith H. Thomas P. Wierda D. (2009). Immunogenicity of biologically-derived therapeutics: Assessment and interpretation of nonclinical safety studies. Regul Toxicol Pharmacol 54, 164–82.
21. Randolph J. F. Stokol T. Scarlett J. M. MacLeod J. N. (1999). Comparison of biological activity and safety of recombinant canine erythropoietin with that of recombinant human erythropoietin in clinically normal dogs. Am J Vet Res 60, 636–42.
22. Rohde C. M. Markiewicz V. Thibault S. Besteman E. Clarke D. W. Perry R. Leach M. W. (2012). Impact of dosing frequency on anaphylaxis in cynomolgus monkeys administered TRU-015, an anti-CD20 SMIP™ therapeutic. Poster presented at the 2012 Society of Toxicology Annual Meeting, San Francisco, California, March 11–15.
23. Rosenberg A. S. (2006). Effects of protein aggregates: An immunologic perspective. AAPS J 8, 501–7.
24. Schellekens H. (2002). Immunogenicity of therapeutic proteins: Clinical implications and future prospects. Clin Ther 24, 1720–40.
25. Stebbings R. Findlay L. Edwards C. Eastwood D. Bird C. North D. Mistry Y. Dilger P. Liefooghe E. Cludts I. Fox B. Tarrant G. Robinson J. Meager T. Dolman C. Thorpe S. J. Bristow A. Wadhwa M. Thorpe R. Poole S. (2007). “Cytokine storm” in the phase I trial of monoclonal antibody TGN1412: Better understanding the causes to improve preclinical testing of immunotherapeutics. J Immunol 179, 3325–31.
26. Suntharalingam G. Perry M. R. Ward S. Brett S. J. Castello-Cortes A. Brunner M. D. Panoskaltsis N. (2006). Cytokine storm in a Phase 1 trial of the anti-CD28 monoclonal antibody TGN1412. N Engl J Med 355, 1018–28.
27. Verhelst D. Rossert J. Casadevall N. Krüger A. Eckardt K. U. MacDougall I. C. (2004). Treatment of erythropoietin-induced pure red cell aplasia: A retrospective study. Lancet 363, 1768–71.
28. Weinbauer G. F. Fuchs A. Niehaus M. Luetjens C. M. (2011). The enhanced pre- and postnatal study for nonhuman primates: Update and perspectives. Birth Defects Res C Embryo Today 93, 324–33.