Scientific considerations for complex drugs in light of established and emerging regulatory guidance

Annals of the New York Academy of Sciences

Volumn 1276, Issue 1, 2012, Pages 26-36

  Holloway, Chris ^a   Mueller Berghaus, Jan ^b   Lima, Beatriz Silva ^c   Lee, Sau Larry ^d   Wyatt, Janet S ^e   Nicholas, J Michael ^f   Crommelin, Daan J A ^g  

^a ERA Consulting Group   (Germany)

^b PAUL EHRLICH INSTITUT   (Germany)

^c UNIVERSITY OF LISBON   (Portugal)

^d CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

^e Institute of Pediatric Nursing   (United States)

^f TEVA PHARMACEUTICALS   (Israel)

^g UTRECHT UNIVERSITY   (Netherlands)

Author keywords

Biosimilars; Complex drug; Follow on biologics; NBCD

Indexed keywords

CALCITONIN; GLATIRAMER; IRON SACCHARATE; MONOCLONAL ANTIBODY; NANOPARTICLE;

ARTICLE; CONSUMER; DRUG FORMULATION; DRUG INDUSTRY; DRUG LEGISLATION; DRUG MARKETING; DRUG SAFETY; KNOWLEDGE; NONHUMAN; PHARMACEUTICAL EQUIVALENCE; PHYSICAL CHEMISTRY; PHYSICIAN; QUALITY CONTROL;

EID: 84871527002     PISSN: 00778923     EISSN: 17496632     Source Type: Book Series    
DOI: 10.1111/j.1749-6632.2012.06811.x     Document Type: Article

References (42)

1. Mayeno, A.N., F. Lin, C.S. Foote, et al. 1990. Characterization of "peak E, "a novel amino acid associated with eosinophilia-myalgia syndrome. Science 250: 1707-1708.
2. Wadhwa, M., A.L. Hjelm Skog, C. Bird, et al. 1999. Immunogenicity of granulocyte-macrophage colony, stimulating factor (GM-CSF) products in patients undergoing combination therapy with GM-CSF. Clin. Cancer Res. 5: 1353-1361.
3. Boven, K., S. Stryker, J. Knight, et al. 2005. The increased incidence of pure red cell aplasia with an Eprex formulation in uncoated rubber stopper syringes. Kidney Int. 67: 2346-2353.
4. Directive 2004/27/EC of the European Parliament and of the Council of 31 March 2004 amending Directive 2001/83/EC on the Community code relating to medicinal products for human use.
5. The Patient Protection and Affordable Care Act (PPAC Act) of 23 March 2010, also referred to in the context of amendments to the Public Health Service Act (PHS Act) as the Biologics Price Competition and Innovation Act (BPCI Act) of 2009.
6. EMEA/190896/2006: Questions and Answers on Recommendation for Refusal of Marketing Application for Alpheon, 28 June 2006.
7. EMEA/CHMP/70349/2008: Withdrawal Assessment Report for Insulin Human Long Marvel.
8. EMA/287731/2011: Questions and Answers, Withdrawal of the marketing authorisation application for Epostim (epoetin alfa), 14 April 2011.
9. EMEA/334517/2009: CHMP (Withdrawal) Assessment Report for Biferonex, July 2009.
10. EMA/CHMP/SWP/100094/2011: Reflection paper on non-clinical studies for generic nanoparticle iron medinal product applications, 17 March 2011.
11. Clark, C. Are all parenteral iron-sucrose preparations identical or just similar Hospital Pharmacy Europe, 58: Oct 2011, reporting results from Toblli, J.E., G. Cao, J. Giani, et al. Different effects of European iron sucrose similar preparations and originator iron sucrose on nitrosative stress, apoptosis, oxidative stress, biochemical and inflammatory markers in rats. XLVIII ERA EDTA Congress 2011; SuO028.
12. Rottembourg, J., A. Kadri, E. Leonard, et al. 2011. Do two intravenous iron sucrose preparations have the same efficacy Nephrol. Dial. Transplant. 26: 3262-3267.
13. EMEA/CHMP/79769/2006: Reflection Paper on Nanotechnology-Based Medicinal Products for Human Use, 29 June 2006.
14. EMA/CHMP/BMWP/652000/2010: Guideline on similar biological medicinal products containing interferon beta (DRAFT).
15. EMA 2001/83/EC/2001: Directive of the European Parliament and of the Council of 6 November 2001 on the Community Code Relating to Medicinal Products for Human Use.
16. European Medicines Agency (EMA), Committee for Medicinal Products for Human Use (CHMP). 2012. Guideline on similar biological medicinal products containing monoclonal antibodies-non-clinical and clinical issues (403543). Retrieved from website: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2012/06/WC500128686.pdf.
17. Toblli, J.E., G. Cao, L. Oliveri & M. Angerosa 2012. Comparison of oxidative stress and inflammation induced by different intravenous iron sucrose similar preparations in a rat model, Inflamm. Allergy-Drug Targets 11: 66-78.
18. MacNeil 2009. WIREs Nanomed Nanobiotechnol 2009 1: 264-271. John Wiley & Sons, Inc.
19. Reflection paper on the data requirements for intravenous liposomal products developed with reference to an innovator liposomal product. Draft, EMA/CHMP/806058/2009, http://www.ema.europa.eu/docs.
20. Reflection paper on non-clinical studies for generic nanoparticle iron medicinal product applications. March 2011, EMA/CHMP/SWP/100094/2011, http://www.ema.europa.eu/docs.
21. Orange book: approved drug products with therapeutic equivalence evaluations. Available at http://www.accessdata.fda.gov/scripts/cder/ob/default.cfm (2009). Accessed 17 June 2012.
22. Lee, S.L., L.X. Yu, B. Cai, et al. 2010. Scientific considerations for generic synthetic salmon calcitonin nasal spray products. AAPS J 13: 14-19.
23. Brown, J.R. & B.S. Hartley 1966. Location of disulphide bridges by diagonal paper electrophoresis. The disulphide bridges of bovine chymotrypsinogen A. Biochem J. 101: 214-228.
24. Creighton, T.E. 1997. Proteins: structure and molecular properties 2nd ed. W. H. Freeman and Company. New York
25. Kozono, T., M. Hirata, K. Endo, et al. 1992. A chimeric analog of human and salmon calcitonin eliminates antigenicity and reduces gastrointestinal disturbances. Endocrinol. 131: 2885-2890.
26. Singer, F.R. 1991. Clinical efficacy of salmon calcitonin in Paget's disease of bone. Calcif. Tissue Int. 49: S7-S8.
27. Rojanasathit, S., E. Rosenberg & J.G. Haddad 1974. Paget's bone disease: response to human calcitonin in patients resistant to salmon calcitonin. Lancet 2: 1412-1415.
28. Schellekens, H. 2005. Factors influencing the immunogenicity of therapeutic proteins. Nephrol. Dial. Transplant. 20: vi3-9.
29. De Groot, A.S. & D.W. Scott Immunogenicity of protein therapeutics. Trends Immunol. 11: 482-490.
30. Windisch, V., F. Deluccia, L. Duhau, et al. 1997. Degradation pathways of salmon calcitonin in aqueous solution. J. Pharm. Sci. 86: 359-364.
31. Bocian, W., J. Sitkowski, A. Tarnowska, et al. 2008. Direct insight into insulin aggregation by 2D NMR complemented by PFGSE NMR. Proteins. 71: 1057-1065.
32. Kamberi, M., P. Chung, R. DeVas, et al. 2004. Analysis of non-covalent aggregation of synthetic hPTH (1-34) by size exclusion chromatography and the importance of suppression of non-specific interactions for a precise quantitation. J. Chromatogr. B Technol. Biomed. Life Sci. 810: 151-155.
33. Lebowitz, J., M.S. Lewis & P. Schuck 2002. Modem analytical ultracentrifugation in protein science: tutorial review. Protein Sci. 11: 2067-2079.
34. Levin, S. 1991. Field flow fractionation in biomedical analysis. Biomed. Chromatogr. 5: 133-137.
35. Pattnaik, P. 2005. Surface plasmon resonance: applications in understanding receptor-ligand interaction. Appl. Biochem. Biotechnol. 126: 79-92.
36. Bruylants, G., J. Wouters & C. Michaux 2005. Differential scanning calorimetry in life science: thermodynamics, stability, molecular recognition and application in drug design. Curr. Med. Chem. 12: 2011-2020.
37. Hudson, F.M. & N.H. Andersen 2004. Exenatide: NMR/CD evaluation of the medium dependence of conformation and aggregation. State. Biopolymers 76: 298-308.
38. Chen, W.L., W.T. Liu, M.C. Yang, et al. 2006. A novel conformation-dependent monoclonal antibody specific to the native structure of beta-lactoglobulin and its application. J. Dairy Sci. 89: 912-921.
39. Costantino, H.R., H. Culley, L. Chen, et al. 2009. Development of calcitonin salmon nasal spray: similarity of peptide formulated in chlorobutanol compared to benzalkonium chloride as preservative. J. Pharm. Sci. 98: 3691-3706.
40. Ball, D., J. Blanchard, D. Jacobson-Kram, et al. 2007. Development of safety qualification thresholds and their use in orally inhaled and nasal drug product evaluation. Toxicol. Sci. 97: 226-236.
41. Norwood, D.L., D. Paskiet, M. Ruberto, et al. 2008. Best practices for extractables and leachables in orally inhaled and nasal drug products: an overview of the PQRI recommendations. Pharm. Res. 25: 727-739.
42. Helmick, C.G., et al. 2008. Estimates of the prevalence of arthritis and other rheumatic conditions in the United States. Part I. Arthritis Rheum. 58: 15-25. doi: 10.1002/art.23177.