Scientific and Regulatory Considerations for Generic Complex Drug Products Containing Nanomaterials

AAPS Journal

Volumn 19, Issue 3, 2017, Pages 619-631

  Zheng, Nan ^a   Sun, Dajun D ^a   Zou, Peng ^a   Jiang, Wenlei ^a  

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

Author keywords

equivalence; generic; nanomaterial; nanotechnology

Indexed keywords

CARBOHYDRATE DERIVATIVE; GENERIC DRUG; IRON DERIVATIVE; LIPOSOME; NANOMATERIAL; NANOTUBE;

ANALYTIC METHOD; ARTICLE; COMPLEX FORMATION; DRUG PROTEIN BINDING; EUROPEAN MEDICINES AGENCY; FOOD AND DRUG ADMINISTRATION; HUMAN; IN VITRO STUDY; IN VIVO STUDY; NANOEMULSION; PHARMACEUTICAL EQUIVALENCE; PHYSICAL CHEMISTRY; DRUG CONTROL; NANOTECHNOLOGY; ORAL DRUG ADMINISTRATION; PARENTERAL DRUG ADMINISTRATION;

ADMINISTRATION, ORAL; DRUG AND NARCOTIC CONTROL; DRUGS, GENERIC; INFUSIONS, PARENTERAL; NANOSTRUCTURES; NANOTECHNOLOGY;

EID: 85010815490     PISSN: None     EISSN: 15507416     Source Type: Journal    
DOI: 10.1208/s12248-017-0044-1     Document Type: Article

References (87)

1. Constantino T. IMS health forecasts global drug spending to increase 30 percent by 2020, to $1.4 trillion, as medicine use gap narrows
2. FDA. Orange book: approved drug products with therapeutic equivalence evaluations. 36th ed. pp. vii. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/UCM071436.pdf2016.
3. FDA. Guidance for Industry: Bioequivalence Studies with Pharmacokinetic Endpoints for Drugs Submitted under an ANDA.
4. FDA. Guidance for Industry: Considering Whether an FDA-Regulated Product Involves the Application of Nanotechnology. http://www.fda.gov/downloads/RegulatoryInformation/Guidances/UCM401695.pdf2014.
5. FDA. Guidance for Industry: Liposome Drug Products - chemistry, manufacturing, and controls; human pharmacokinetics and bioavailability; and labeling documentation. http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm070570.pdf2015.
6. FDA. Product-specific recommendations for generic drug development. http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm075207.htm.
7. Zhang L, Wang S, Zhang M, Sun J. Nanocarriers for oral drug delivery. J Drug Target. 2013;21(6):515–27.
8. Ensign LM, Cone R, Hanes J. Oral drug delivery with polymeric nanoparticles: the gastrointestinal mucus barriers. Adv Drug Deliv Rev. 2012;64(6):557–70.
9. Shegokar R, Müller RH. Nanocrystals: industrially feasible multifunctional formulation technology for poorly soluble actives. Int J Pharm. 2010;399(1):129–39.
10. Mishra AK, Vachon MG, Guivarc’h P-H, Snow RA, Pace GW. IDD technology: oral delivery of water insoluble drugs using phospholipid-stabilized microparticulate IDD formulations. In: Rathbone MJ, Hadgraft J, Roberts MS, editors. Modified-release drug delivery technology. New York: Marcel Dekker; 2002. p. 151–75.
11. Kesisoglou F, Panmai S, Wu Y. Nanosizing—oral formulation development and biopharmaceutical evaluation. Adv Drug Deliv Rev. 2007;59(7):631–44.
12. Deschamps B, Musaji N, Gillespie JA. Food effect on the bioavailability of two distinct formulations of megestrol acetate oral suspension. Int J Nanomedicine. 2009;4:185.
13. Tierney TB, Guo Y, Beloshapkin S, Rasmuson ÅC, Hudson SP. Investigation of the particle growth of fenofibrate following antisolvent precipitation and freeze–drying. Cryst Growth Des. 2015;15(11):5213–22.
14. Wu Y, Loper A, Landis E, Hettrick L, Novak L, Lynn K, et al. The role of biopharmaceutics in the development of a clinical nanoparticle formulation of MK-0869: a beagle dog model predicts improved bioavailability and diminished food effect on absorption in human. Int J Pharm. 2004;285(1):135–46.
15. Purvis T, Overhoff KA, Sinswat P, Williams RO. Immunosuppressant drugs. In: Williams RO, Taft DR, McConville JT, editors. Advanced drug formulation design to optimize therapeutic outcomes. Boca Raton, FL: CRC Press, Taylor & Francis Group; 2007.
16. Gao Z-G, Choi H-G, Shin H-J, Park K-M, Lim S-J, Hwang K-J, et al. Physicochemical characterization and evaluation of a microemulsion system for oral delivery of cyclosporin A. Int J Pharm. 1998;161(1):75–86.
17. Weissig V, Pettinger TK, Murdock N. Nanopharmaceuticals (part 1): products on the market. Int J Nanomedicine. 2014;9:4357.
18. Desai PP, Date AA, Patravale VB. Overcoming poor oral bioavailability using nanoparticle formulations—opportunities and limitations. Drug Discov Today Technol. 2012;9(2):e87–95.
19. Kalepu S, Nekkanti V. Insoluble drug delivery strategies: review of recent advances and business prospects. Acta Pharm Sin B. 2015;5(5):442–53.
20. Wu Y, Petrochenko P, Chen L, Wong SY, Absar M, Choi S, et al. Core size determination and structural characterization of intravenous iron complexes by cryogenic transmission electron microscopy. Int J Pharm. 2016;505(1–2):167–74. doi:10.1016/j.ijpharm.2016.03.029.
21. Jahn MR, Andreasen HB, Futterer S, Nawroth T, Schunemann V, Kolb U, et al. A comparative study of the physicochemical properties of iron isomaltoside 1000 (Monofer), a new intravenous iron preparation and its clinical implications. Eur J Pharm Biopharm: Off J Arbeitsgemeinschaft Pharm Verfahrenstec eV. 2011;78(3):480–91. doi:10.1016/j.ejpb.2011.03.016.
22. Geisser P, Burckhardt S. The pharmacokinetics and pharmacodynamics of iron preparations. Pharmaceutics. 2011;3(1):12–33. doi:10.3390/pharmaceutics3010012.
23. Beshara S, Lundqvist H, Sundin J, Lubberink M, Tolmachev V, Valind S, et al. Pharmacokinetics and red cell utilization of iron(III) hydroxide-sucrose complex in anaemic patients: a study using positron emission tomography. Br J Haematol. 1999;104(2):296–302.
24. Sanofi Aventis US. FERRLECIT Label. http://www.accessdata.fda.gov/drugsatfda_docs/label/2011/020955s013s015lbl.pdf2011.
25. Serum iron test. https://medlineplus.gov/ency/article/003488.htm
26. Van Wyck D, Anderson J, Johnson K. Labile iron in parenteral iron formulations: a quantitative and comparative study. Nephrol Dial Transplant. 2004;19(3):561–5.
27. Danielson BG, Salmonson T, Derendorf H, Geisser P. Pharmacokinetics of iron(III)-hydroxide sucrose complex after a single intravenous dose in healthy volunteers. Arzneimittelforschung. 1996;46(6):615–21.
28. Seligman PA, Dahl NV, Strobos J, Kimko HC, Schleicher RB, Jones M, et al. Single-dose pharmacokinetics of sodium ferric gluconate complex in iron-deficient subjects. Pharmacotherapy. 2004;24(5):574–83.
29. Theil EC. Ferritin: structure, gene regulation, and cellular function in animals, plants, and microorganisms. Annu Rev Biochem. 1987;56:289–315. doi:10.1146/annurev.bi.56.070187.001445.
30. Andrews SC, Arosio P, Bottke W, Briat JF, von Darl M, Harrison PM, et al. Structure, function, and evolution of ferritins. J Inorg Biochem. 1992;47(3–4):161–74.
31. Provenzano R, Schiller B, Rao M, Coyne D, Brenner L, Pereira BJG. Ferumoxytol as an intravenous iron replacement therapy in hemodialysis patients. Clin J Am Soc Nephro. 2009;4(2):386–93. doi:10.2215/Cjn.02840608.
32. Jacobs EM, Hendriks JC, van Tits BL, Evans PJ, Breuer W, Liu DY, et al. Results of an international round robin for the quantification of serum non-transferrin-bound iron: need for defining standardization and a clinically relevant isoform. Anal Biochem. 2005;341(2):241–50. doi:10.1016/j.ab.2005.03.008.
33. Pai AB, Boyd AV, McQuade CR, Harford A, Norenberg JP, Zager PG. Comparison of oxidative stress markers after intravenous administration of iron dextran, sodium ferric gluconate, and iron sucrose in patients undergoing hemodialysis. Pharmacotherapy. 2007;27(3):343–50. doi:10.1592/phco.27.3.343.
34. Chouly C, Pouliquen D, Lucet I, Jeune JJ, Jallet P. Development of superparamagnetic nanoparticles for MRI: effect of particle size, charge and surface nature on biodistribution. J Microencapsul. 1996;13(3):245–55.
35. Ellison HL, Hazel F. Influence of concentration and age on some colloidal properties of ferric chloride solutions. J Phys Chem-Us. 1935;39(6):829–35. doi:10.1021/J150366a011.
36. Whitehead TH. The complex compound theory of colloidal oxides. Chem Rev. 1937;21(1):113–28. doi:10.1021/Cr60068a004.
37. Lawrence R. Development and comparison of iron dextran products. PDA J Pharm Sci Technol. 1998;52(5):190–7.
38. Yang Y, Shah RB, Faustino PJ, Raw A, Yu LX, Khan MA. Thermodynamic stability assessment of a colloidal iron drug product: sodium ferric gluconate. J Pharm Sci. 2010;99(1):142–53. doi:10.1002/jps.21806.
39. Hasan DM, Amans M, Tihan T, Hess C, Guo Y, Cha S, et al. Ferumoxytol-enhanced MRI to image inflammation within human brain arteriovenous malformations: a pilot investigation. Transl Stroke Res. 2012;3(Supplement 1):166–73. doi:10.1007/s12975-012-0172-y.
40. Agarwal R. Transferrin saturation with intravenous irons: an in vitro study. Kidney Int. 2004;66(3):1139–44.
41. Toblli JE, Cao G, Oliveri L, Angerosa M. Comparison of oxidative stress and inflammation induced by different intravenous iron sucrose similar preparations in a rat model. Inflamm Allergy Drug Targets. 2012;11(1):66–78.
42. Research Funding Announcement (RFA-FD-13-017) Development of bio-relevant assay to determine labile iron in the parenteral iron complex product. The U.S. Food and Drug Administration (https://grants.nih.gov/grants/guide/rfa-files/RFA-FD-13-017.html). 2013.
43. Research Funding Announcement (RFA-FD-14-019) Evaluation of iron species in healthy subjects treated with generic and reference sodium ferric gluconate. The U.S. Food and Drug Administration (http://grants.nih.gov/grants/guide/rfa-files/RFA-FD-14-019.html). 2014.
44. Mrozek E, Rhoades CA, Allen J, Hade EM, Shapiro CL. Phase I trial of liposomal encapsulated doxorubicin (Myocet; D-99) and weekly docetaxel in advanced breast cancer patients. Ann Oncol: Off J Eur Soc Med Oncol / ESMO. 2005;16(7):1087–93. doi:10.1093/annonc/mdi209.
45. Akbarzadeh A, Rezaei-Sadabady R, Davaran S, Joo SW, Zarghami N, Hanifehpour Y, et al. Liposome: classification, preparation, and applications. Nanoscale Res Lett. 2013;8(1):102.
46. Zheng N JW, Lionberger R, Yu L. Bioequivalence for Liposomal Drug Products. FDA Bioequivalence Standards 2014.
47. Schellekens H, Klinger E, Muhlebach S, Brin JF, Storm G, Crommelin DJ. The therapeutic equivalence of complex drugs. Regul Toxicol Pharmacol: RTP. 2011;59(1):176–83. doi:10.1016/j.yrtph.2010.09.021.
48. Zhigaltsev IV, Maurer N, Akhong QF, Leone R, Leng E, Wang J, et al. Liposome-encapsulated vincristine, vinblastine and vinorelbine: a comparative study of drug loading and retention. J Control Release: Off J Control Release Soc. 2005;104(1):103–11. doi:10.1016/j.jconrel.2005.01.010.
49. Drummond DC, Noble CO, Hayes ME, Park JW, Kirpotin DB. Pharmacokinetics and in vivo drug release rates in liposomal nanocarrier development. J Pharm Sci. 2008;97(11):4696–740.
50. Maeda H. The enhanced permeability and retention (EPR) effect in tumor vasculature: the key role of tumor-selective macromolecular drug targeting. Adv Enzym Regul. 2001;41:189–207.
51. Chowdhary RK, Shariff I, Dolphin D. Drug release characteristics of lipid based benzoporphyrin derivative. J Pharm Pharm Sci. 2003;6(1):13–9.
52. Novartis. VISUDYNE Label. http://dailymed.nlm.nih.gov/dailymed/lookup.cfm?setid=31512723-9ff0-4e18-aa3a-55ab833038c6 2012.
53. Richter AM, Waterfield E, Jain AK, Canaan AJ, Allison BA, Levy JG. Liposomal delivery of a photosensitizer, benzoporphyrin derivative monoacid ring A (BPD), to tumor tissue in a mouse tumor model. Photochem Photobiol. 1993;57(6):1000–6.
54. Csuhai E, Kangarlou S, Xiang TX, Ponta A, Bummer P, Choi D, et al. Determination of key parameters for a mechanism‐based model to predict doxorubicin release from actively loaded liposomes. J Pharm Sci. 2015;104(3):1087–98.
55. Fugit KD, Xiang T-X, Choi DH, Kangarlou S, Csuhai E, Bummer PM, et al. Mechanistic model and analysis of doxorubicin release from liposomal formulations. J Control Release: Off J Control Release Soc. 2015;217:82–91.
56. Elsadek B, Kratz F. Impact of albumin on drug delivery—new applications on the horizon. J Control Rel: Off J Control Release Soc. 2012;157(1):4–28. doi:10.1016/j.jconrel.2011.09.069.
57. Abraxis BioScience. ABRAXANE Label. http://www.accessdata.fda.gov/drugsatfda_docs/label/2012/021660s031lbl.pdf 2012.
58. Hawkins MJ, Soon-Shiong P, Desai N. Protein nanoparticles as drug carriers in clinical medicine. Adv Drug Deliv Rev. 2008;60(8):876–85. doi:10.1016/j.addr.2007.08.044.
59. Abraxis BioScience. Abraxane. http://www.fda.gov/ohrms/dockets/ac/06/slides/2006-4235S2-02-01-FDAAbraxane.ppt2006.
60. Sparreboom A, Scripture CD, Trieu V, Williams PJ, De T, Yang A, et al. Comparative preclinical and clinical pharmacokinetics of a cremophor-free, nanoparticle albumin-bound paclitaxel (ABI-007) and paclitaxel formulated in cremophor (Taxol). Clin Cancer Res: Off J Am Assoc Cancer Res. 2005;11(11):4136–43. doi:10.1158/1078-0432.CCR-04-2291.
61. Desai N, Trieu V, Yao Z, Louie L, Ci S, Yang A, et al. Increased antitumor activity, intratumor paclitaxel concentrations, and endothelial cell transport of cremophor-free, albumin-bound paclitaxel, ABI-007, compared with cremophor-based paclitaxel. Clin Cancer Res: Off J Am Assoc Cancer Res. 2006;12(4):1317–24.
62. Chen N, Brachmann C, Liu X, Pierce DW, Dey J, Kerwin WS, et al. Albumin-bound nanoparticle (nab) paclitaxel exhibits enhanced paclitaxel tissue distribution and tumor penetration. Cancer Chemother Pharmacol. 2015;76(4):699–712.
63. EMA. Assessment Report for Abraxane-Annex 1 Summary of Product Characteristics. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/000778/WC500020435.pdf2007.
64. Sparreboom A, van Zuylen L, Brouwer E, Loos WJ, de Bruijn P, Gelderblom H, et al. Cremophor EL-mediated alteration of paclitaxel distribution in human blood: clinical pharmacokinetic implications. Cancer Res. 1999;59(7):1454–7.
65. Gardner ER, Dahut W, Figg WD. Quantitative determination of total and unbound paclitaxel in human plasma following Abraxane treatment. J Chromatogr B Anal Technol Biomed Life Sci. 2008;862(1–2):213–8. doi:10.1016/j.jchromb.2007.12.013.
66. J&J PRD. DOXIL Label: http://www.accessdata.fda.gov/drugsatfda_docs/label/2015/050718s048lbl.pdf.
67. Gelgene. Citizen Petition from Arnold & Porter LLP (Celgene): Table 4 and Table 5. 2015; Available from: https://www.regulations.gov/#!documentDetail;D=FDA-2015-P-0732-0001.
68. Paal K, Shkarupin A. Paclitaxel binding to the fatty acid-induced conformation of human serum albumin—automated docking studies. Bioorg Med Chem. 2007;15(24):7568–75. doi:10.1016/j.bmc.2007.09.006.
69. Valery C, Artzner F, Robert B, Gulick T, Keller G, Grabielle-Madelmont C, et al. Self-association process of a peptide in solution: from beta-sheet filaments to large embedded nanotubes. Biophys J. 2004;86(4):2484–501. doi:10.1016/S0006-3495(04)74304-0.
70. Cherif-Cheikh R. Sustained release of peptides from pharmaceutical compositions. Google Patents; 1997.
71. Pouget E, Fay N, Dujardin E, Jamin N, Berthault P, Perrin L, et al. Elucidation of the self-assembly pathway of lanreotide octapeptide into beta-sheet nanotubes: role of two stable intermediates. J Am Chem Soc. 2010;132(12):4230–41. doi:10.1021/ja9088023.
72. Valery C, Paternostre M, Robert B, Gulik-Krzywicki T, Narayanan T, Dedieu JC, et al. Biomimetic organization: octapeptide self-assembly into nanotubes of viral capsid-like dimension. Proc Natl Acad Sci U S A. 2003;100(18):10258–62. doi:10.1073/pnas.1730609100.
73. Valery C, Pouget E, Pandit A, Verbavatz JM, Bordes L, Boisde I, et al. Molecular origin of the self-assembly of lanreotide into nanotubes: a mutational approach. Biophys J. 2008;94(5):1782–95. doi:10.1529/biophysj.107.108175.
74. Tarabout C, Roux S, Gobeaux F, Fay N, Pouget E, Meriadec C, et al. Control of peptide nanotube diameter by chemical modifications of an aromatic residue involved in a single close contact. Proc Natl Acad Sci U S A. 2011;108(19):7679–84. doi:10.1073/pnas.1017343108.
75. Gobeaux F, Fay N, Tarabout C, Meriadec C, Meneau F, Ligeti M, et al. Structural role of counterions adsorbed on self-assembled peptide nanotubes. J Am Chem Soc. 2012;134(1):723–33. doi:10.1021/ja210299g.
76. Badaire S, Poulin P, Maugey M, Zakri C. In situ measurements of nanotube dimensions in suspensions by depolarized dynamic light scattering. Langmuir. 2004;20(24):10367–70.
77. Wang SR, Liang ZY, Wang B, Zhang C. Statistical characterization of single-wall carbon nanotube length distribution. Nanotechnology. 2006;17(3):634–9. doi:10.1088/0957-4484/17/3/003.
78. Lee SL, Saluja B, Garcia-Arieta A, Santos GM, Li Y, Lu S, et al. Regulatory considerations for approval of generic inhalation drug products in the US, EU, Brazil, China, and India. AAPS J. 2015;17(5):1285–304. doi:10.1208/s12248-015-9787-8.
79. Lee SL, Yu LX, Cai B, Johnsons GR, Rosenberg AS, Cherney BW, et al. Scientific considerations for generic synthetic salmon calcitonin nasal spray products. AAPS J. 2011;13(1):14–9. doi:10.1208/s12248-010-9242-9.
80. FDA. FDA/CDER response to Citizen Petition from Ortho-Biotech Products, L.P. http://www.regulations.gov/#!docketDetail;D=FDA-2009-P-02162013.
81. Crommelin DJA. Challenges for Non-Biological Complex Drugs (NBCDs). http://www.fda.gov/downloads/ForIndustry/UserFees/GenericDrugUserFees/UCM398889.pdf2014.
82. EMA. Reflection paper on the data requirements for intravenous liposomal products developed with reference to an innovator liposomal product. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2013/03/WC500140351.pdf2013.
83. EMA. Reflection paper on the data requirements for intravenous iron-based nano-colloidal products developed with reference to an innovator medicinal product. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2015/03/WC500184922.pdf2015.
84. FDA. Product Specific Bioequivalence Recommendation: Doxorubicin Hydrochloride. http://www.fda.gov/downloads/Drugs/…/Guidances/UCM199635.pdf2014.
85. Rottembourg J, Kadri A, Leonard E, Dansaert A, Lafuma A. Do two intravenous iron sucrose preparations have the same efficacy? Nephrol Dial Transplant. 2011;26(10):3262–7. doi:10.1093/ndt/gfr024.
86. Stein J, Dignass A, Chow KU. Clinical case reports raise doubts about the therapeutic equivalence of an iron sucrose similar preparation compared with iron sucrose originator. Curr Med Res Opin. 2012;28(2):241–3. doi:10.1185/03007995.2011.651527.
87. Crommelin DJ, Shah VP, Klebovich I, McNeil SE, Weinstein V, Fluhmann B, et al. The similarity question for biologicals and non-biological complex drugs. Eur J Pharm Sci. 2015;76:10–7.