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Nanoparticle-based drug delivery systems: a commercial and regulatory outlook as the field matures.

Bibliographic reference Ragelle, Héloïse ; Danhier, Fabienne ; Préat, Véronique ; Langer, Robert ; Anderson, Daniel G. Nanoparticle-based drug delivery systems: a commercial and regulatory outlook as the field matures.. In: Expert Opinion on Drug Delivery, Vol. 14, no. 7, p. 851-864
Permanent URL http://hdl.handle.net/2078.1/180580
  1. Paul Evers BCC Research, Nanotechnology in medical applications: the global market (2015)
  2. Venditto Vincent J., Szoka Francis C., Cancer nanomedicines: So many papers and so few drugs!, 10.1016/j.addr.2012.09.038
  3. Sarkar Arindam Basu, Journal of Pharmaceutical Sciences and Pharmacology: A New Perspective, 10.1166/jpsp.2014.1001
  4. Etheridge Michael L., Campbell Stephen A., Erdman Arthur G., Haynes Christy L., Wolf Susan M., McCullough Jeffrey, The big picture on nanomedicine: the state of investigational and approved nanomedicine products, 10.1016/j.nano.2012.05.013
  5. Hafner A, Int J Nanomed, 9, 1005 (2014)
  6. Tibbitt Mark W., Dahlman James E., Langer Robert, Emerging Frontiers in Drug Delivery, 10.1021/jacs.5b09974
  7. Alexander Archie A., Jotterand Fabrice, Market Considerations for Nanomedicines and Theranostic Nanomedicines, Cancer Theranostics (2014) ISBN:9780124077225 p.471-491, 10.1016/b978-0-12-407722-5.00025-6
  8. Wagner Volker, Dullaart Anwyn, Bock Anne-Katrin, Zweck Axel, The emerging nanomedicine landscape, 10.1038/nbt1006-1211
  9. Petros Robby A., DeSimone Joseph M., Strategies in the design of nanoparticles for therapeutic applications, 10.1038/nrd2591
  10. Blanco Elvin, Hsiao Angela, Mann Aman P., Landry Matthew G., Meric-Bernstam Funda, Ferrari Mauro, Nanomedicine in cancer therapy: Innovative trends and prospects, 10.1111/j.1349-7006.2011.01941.x
  11. Bangham A.D., Standish M.M., Watkins J.C., Diffusion of univalent ions across the lamellae of swollen phospholipids, 10.1016/s0022-2836(65)80093-6
  12. Yatvin M., Kreutz W, Horwitz B., Shinitzky M, pH-sensitive liposomes: possible clinical implications, 10.1126/science.7434025
  13. Allen T.M., Chonn A., Large unilamellar liposomes with low uptake into the reticuloendothelial system, 10.1016/0014-5793(87)80506-9
  14. Barenholz Yechezkel (Chezy), Doxil® — The first FDA-approved nano-drug: Lessons learned, 10.1016/j.jconrel.2012.03.020
  15. Gordon Alan N., Fleagle John T., Guthrie David, Parkin David E., Gore Martin E., Lacave Angel J., Recurrent Epithelial Ovarian Carcinoma: A Randomized Phase III Study of Pegylated Liposomal Doxorubicin Versus Topotecan, 10.1200/jco.2001.19.14.3312
  16. Nichols Joseph W., Bae You Han, EPR: Evidence and fallacy, 10.1016/j.jconrel.2014.03.057
  17. Solomon Rebecca, Gabizon Alberto A., Clinical Pharmacology of Liposomal Anthracyclines: Focus on Pegylated Liposomal Doxorubicin, 10.3816/clm.2008.n.001
  18. Gabizon A, Cancer Res, 54, 987 (1994)
  19. Szebeni Janos, Muggia Franco, Gabizon Alberto, Barenholz Yechezkel, Activation of complement by therapeutic liposomes and other lipid excipient-based therapeutic products: Prediction and prevention, 10.1016/j.addr.2011.06.017
  20. Szebeni Janos, Complement activation-related pseudoallergy: A new class of drug-induced acute immune toxicity, 10.1016/j.tox.2005.07.023
  21. Sparreboom A., Comparative Preclinical and Clinical Pharmacokinetics of a Cremophor-Free, Nanoparticle Albumin-Bound Paclitaxel (ABI-007) and Paclitaxel Formulated in Cremophor (Taxol), 10.1158/1078-0432.ccr-04-2291
  22. Desai Neil, Challenges in Development of Nanoparticle-Based Therapeutics, 10.1208/s12248-012-9339-4
  23. Gardner E. R., Dahut W. L., Scripture C. D., Jones J., Aragon-Ching J. B., Desai N., Hawkins M. J., Sparreboom A., Figg W. D., Randomized Crossover Pharmacokinetic Study of Solvent-Based Paclitaxel and nab-Paclitaxel, 10.1158/1078-0432.ccr-07-4592
  24. Henderson I Craig, Bhatia Vinona, Nab-paclitaxel for breast cancer: a new formulation with an improved safety profile and greater efficacy, 10.1586/14737140.7.7.919
  25. Korsmeyer Richard, Critical questions in development of targeted nanoparticle therapeutics, 10.1093/rb/rbw011
  26. Yin Hao, Kanasty Rosemary L., Eltoukhy Ahmed A., Vegas Arturo J., Dorkin J. Robert, Anderson Daniel G., Non-viral vectors for gene-based therapy, 10.1038/nrg3763
  27. Colombo S, Expert Opin Drug Deliv, 1 (2014)
  28. Kanasty Rosemary, Dorkin Joseph Robert, Vegas Arturo, Anderson Daniel, Delivery materials for siRNA therapeutics, 10.1038/nmat3765
  29. Wittrup Anders, Ai Angela, Liu Xing, Hamar Peter, Trifonova Radiana, Charisse Klaus, Manoharan Muthiah, Kirchhausen Tomas, Lieberman Judy, Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown, 10.1038/nbt.3298
  30. Ragelle Héloïse, Colombo Stefano, Pourcelle Vincent, Vanvarenberg Kevin, Vandermeulen Gaëlle, Bouzin Caroline, Marchand-Brynaert Jacqueline, Feron Olivier, Foged Camilla, Préat Véronique, Intracellular siRNA delivery dynamics of integrin-targeted, PEGylated chitosan–poly(ethylene imine) hybrid nanoparticles: A mechanistic insight, 10.1016/j.jconrel.2015.05.274
  31. Akinc Akin, Goldberg Michael, Qin June, Dorkin J Robert, Gamba-Vitalo Christina, Maier Martin, Jayaprakash K Narayanannair, Jayaraman Muthusamy, Rajeev Kallanthottathil G, Manoharan Muthiah, Koteliansky Victor, Röhl Ingo, Leshchiner Elizaveta S, Langer Robert, Anderson Daniel G, Development of Lipidoid–siRNA Formulations for Systemic Delivery to the Liver, 10.1038/mt.2009.36
  32. Kamaly Nazila, Xiao Zeyu, Valencia Pedro M., Radovic-Moreno Aleksandar F., Farokhzad Omid C., Targeted polymeric therapeutic nanoparticles: design, development and clinical translation, 10.1039/c2cs15344k
  33. Bertrand Nicolas, Wu Jun, Xu Xiaoyang, Kamaly Nazila, Farokhzad Omid C., Cancer nanotechnology: The impact of passive and active targeting in the era of modern cancer biology, 10.1016/j.addr.2013.11.009
  34. Danhier Fabienne, Feron Olivier, Préat Véronique, To exploit the tumor microenvironment: Passive and active tumor targeting of nanocarriers for anti-cancer drug delivery, 10.1016/j.jconrel.2010.08.027
  35. Danhier Fabienne, Vroman Benoît, Lecouturier Nathalie, Crokart Nathalie, Pourcelle Vincent, Freichels Hélène, Jérôme Christine, Marchand-Brynaert Jacqueline, Feron Olivier, Préat Véronique, Targeting of tumor endothelium by RGD-grafted PLGA-nanoparticles loaded with Paclitaxel, 10.1016/j.jconrel.2009.08.011
  36. Corbet Cyril, Ragelle Héloïse, Pourcelle Vincent, Vanvarenberg Kévin, Marchand-Brynaert Jacqueline, Préat Véronique, Feron Olivier, Delivery of siRNA targeting tumor metabolism using non-covalent PEGylated chitosan nanoparticles: Identification of an optimal combination of ligand structure, linker and grafting method, 10.1016/j.jconrel.2015.12.020
  37. Graf Nora, Bielenberg Diane R., Kolishetti Nagesh, Muus Christoph, Banyard Jacqueline, Farokhzad Omid C., Lippard Stephen J., αVβ3 Integrin-Targeted PLGA-PEG Nanoparticles for Enhanced Anti-tumor Efficacy of a Pt(IV) Prodrug, 10.1021/nn301148e
  38. Hrkach J., Von Hoff D., Ali M. M., Andrianova E., Auer J., Campbell T., De Witt D., Figa M., Figueiredo M., Horhota A., Low S., McDonnell K., Peeke E., Retnarajan B., Sabnis A., Schnipper E., Song J. J., Song Y. H., Summa J., Tompsett D., Troiano G., Van Geen Hoven T., Wright J., LoRusso P., Kantoff P. W., Bander N. H., Sweeney C., Farokhzad O. C., Langer R., Zale S., Preclinical Development and Clinical Translation of a PSMA-Targeted Docetaxel Nanoparticle with a Differentiated Pharmacological Profile, 10.1126/scitranslmed.3003651
  39. Mamot Christoph, Ritschard Reto, Wicki Andreas, Stehle Gregor, Dieterle Thomas, Bubendorf Lukas, Hilker Christoph, Deuster Stefanie, Herrmann Richard, Rochlitz Christoph, Tolerability, safety, pharmacokinetics, and efficacy of doxorubicin-loaded anti-EGFR immunoliposomes in advanced solid tumours: a phase 1 dose-escalation study, 10.1016/s1470-2045(12)70476-x
  40. Farokhzad Omid C., Langer Robert, Impact of Nanotechnology on Drug Delivery, 10.1021/nn900002m
  41. Dreher Matthew R., Liu Wenge, Michelich Charles R., Dewhirst Mark W., Yuan Fan, Chilkoti Ashutosh, Tumor Vascular Permeability, Accumulation, and Penetration of Macromolecular Drug Carriers, 10.1093/jnci/djj070
  42. Reddy Sai T, van der Vlies André J, Simeoni Eleonora, Angeli Veronique, Randolph Gwendalyn J, O'Neil Conlin P, Lee Leslie K, Swartz Melody A, Hubbell Jeffrey A, Exploiting lymphatic transport and complement activation in nanoparticle vaccines, 10.1038/nbt1332
  43. Sancey L, Lux F, Kotb S, Roux S, Dufort S, Bianchi A, Crémillieux Y, Fries P, Coll J-L, Rodriguez-Lafrasse C, Janier M, Dutreix M, Barberi-Heyob M, Boschetti F, Denat F, Louis C, Porcel E, Lacombe S, Le Duc G, Deutsch E, Perfettini J-L, Detappe A, Verry C, Berbeco R, Butterworth K T, McMahon S J, Prise K M, Perriat P, Tillement O, The use of theranostic gadolinium-based nanoprobes to improve radiotherapy efficacy, 10.1259/bjr.20140134
  44. Maggiorella Laurence, Barouch Gilles, Devaux Corinne, Pottier Agnès, Deutsch Eric, Bourhis Jean, Borghi Elsa, Levy Laurent, Nanoscale radiotherapy with hafnium oxide nanoparticles, 10.2217/fon.12.96
  45. Rathore Anurag S, Winkle Helen, Quality by design for biopharmaceuticals, 10.1038/nbt0109-26
  46. Vauthier Christine, Bouchemal Kawthar, Methods for the Preparation and Manufacture of Polymeric Nanoparticles, 10.1007/s11095-008-9800-3
  47. Dhir Sanjeev, Morrow K. John, Rhinehart R. Russell, Wiesner Theodore, Dynamic optimization of hybridoma growth in a fed-batch bioreactor, 10.1002/(sici)1097-0290(20000120)67:2<197::aid-bit9>3.0.co;2-w
  48. Lakkireddy Harivardhan Reddy, Bazile Didier, Building the design, translation and development principles of polymeric nanomedicines using the case of clinically advanced poly(lactide(glycolide))–poly(ethylene glycol) nanotechnology as a model: An industrial viewpoint, 10.1016/j.addr.2016.08.012
  49. Mühlebach Stefan, Borchard Gerrit, Yildiz Selcan, Regulatory challenges and approaches to characterize nanomedicines and their follow-on similars, 10.2217/nnm.14.189
  50. Vetten Melissa A., Yah Clarence S., Singh Tanusha, Gulumian Mary, Challenges facing sterilization and depyrogenation of nanoparticles: Effects on structural stability and biomedical applications, 10.1016/j.nano.2014.03.017
  51. Zucker Daniel, Andriyanov Alexander V., Steiner Ariel, Raviv Uri, Barenholz Yechezkel, Characterization of PEGylated nanoliposomes co-remotely loaded with topotecan and vincristine: relating structure and pharmacokinetics to therapeutic efficacy, 10.1016/j.jconrel.2011.10.003
  52. Hare JI, Adv Drug Deliv Rev (2016)
  53. Sadrieh N, Conf Present Nanotechnol Impact Heal Regul (2005)
  54. Dobrovolskaia Marina A., McNeil Scott E., Understanding the correlation between in vitro and in vivo immunotoxicity tests for nanomedicines, 10.1016/j.jconrel.2013.05.025
  55. Tinkle Sally, McNeil Scott E., Mühlebach Stefan, Bawa Raj, Borchard Gerrit, Barenholz Yechezkel Chezy, Tamarkin Lawrence, Desai Neil, Nanomedicines: addressing the scientific and regulatory gap : Nanomedicines, 10.1111/nyas.12403
  56. Le Tourneau Christophe, Lee J. Jack, Siu Lillian L., Dose Escalation Methods in Phase I Cancer Clinical Trials, 10.1093/jnci/djp079
  57. Cook David, Brown Dearg, Alexander Robert, March Ruth, Morgan Paul, Satterthwaite Gemma, Pangalos Menelas N., Lessons learned from the fate of AstraZeneca's drug pipeline: a five-dimensional framework, 10.1038/nrd4309
  58. Arrowsmith John, Miller Philip, Trial Watch: Phase II and Phase III attrition rates 2011–2012, 10.1038/nrd4090
  59. Seymour L., Ivy S. P., Sargent D., Spriggs D., Baker L., Rubinstein L., Ratain M. J., Le Blanc M., Stewart D., Crowley J., Groshen S., Humphrey J. S., West P., Berry D., The Design of Phase II Clinical Trials Testing Cancer Therapeutics: Consensus Recommendations from the Clinical Trial Design Task Force of the National Cancer Institute Investigational Drug Steering Committee, 10.1158/1078-0432.ccr-09-3287
  60. Sertkaya Aylin, Wong Hui-Hsing, Jessup Amber, Beleche Trinidad, Key cost drivers of pharmaceutical clinical trials in the United States, 10.1177/1740774515625964
  61. Eifler Aaron C., Thaxton C. Shad, Nanoparticle Therapeutics: FDA Approval, Clinical Trials, Regulatory Pathways, and Case Study, Methods in Molecular Biology (2011) ISBN:9781617790515 p.325-338, 10.1007/978-1-61779-052-2_21
  62. Bobo Daniel, Robinson Kye J., Islam Jiaul, Thurecht Kristofer J., Corrie Simon R., Nanoparticle-Based Medicines: A Review of FDA-Approved Materials and Clinical Trials to Date, 10.1007/s11095-016-1958-5
  63. U.S. Department of Health and Human Services, FDA, Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, Guidance for industry ANDA submissions – content and format of abbreviated new drug applications (2014)
  64. Crommelin Daan J.A., Metselaar Josbert M., Storm Gert, Liposomes: The Science and the Regulatory Landscape, Non-Biological Complex Drugs (2015) ISBN:9783319162409 p.77-106, 10.1007/978-3-319-16241-6_3
  65. Hehenberger M, Nanomedicine: science, business, and impact (2016)
  66. Sainz Vanessa, Conniot João, Matos Ana I., Peres Carina, Zupanǒiǒ Eva, Moura Liane, Silva Liana C., Florindo Helena F., Gaspar Rogério S., Regulatory aspects on nanomedicines, 10.1016/j.bbrc.2015.08.023
  67. Crommelin Daan J.A., Shah Vinod P., Klebovich Imre, McNeil Scott E., Weinstein Vera, Flühmann Beat, Mühlebach Stefan, de Vlieger Jon S.B., The similarity question for biologicals and non-biological complex drugs, 10.1016/j.ejps.2015.04.010
  68. Schellekens Huub, Stegemann Sven, Weinstein Vera, de Vlieger Jon S. B., Flühmann Beat, Mühlebach Stefan, Gaspar Rogério, Shah Vinod P., Crommelin Daan J. A., How to Regulate Nonbiological Complex Drugs (NBCD) and Their Follow-on Versions: Points to Consider, 10.1208/s12248-013-9533-z
  69. Holloway Chris, Mueller-Berghaus Jan, Lima Beatriz Silva, Lee Sau Larry, Wyatt Janet S., Nicholas J. Michael, Crommelin Daan J.A., Scientific considerations for complex drugs in light of established and emerging regulatory guidance : Scientific considerations for complex drugs, 10.1111/j.1749-6632.2012.06811.x
  70. Ehmann Falk, Sakai-Kato Kumiko, Duncan Ruth, Pérez de la Ossa Dolores Hernán, Pita Ruben, Vidal Jean-Marc, Kohli Ashish, Tothfalusi Laszlo, Sanh Alan, Tinton Sandrine, Robert Jean-Louis, Silva Lima Beatriz, Amati Marisa Papaluca, Next-generation nanomedicines and nanosimilars: EU regulators’ initiatives relating to the development and evaluation of nanomedicines, 10.2217/nnm.13.68
  71. Committee for Human Medicinal Products (CHMP). European Medicines Agency, Reflection paper on the data requirements for intravenous liposomal products developed with reference to an innovator liposomal producte (2013)
  72. U.S. Department of Health and Human Services, FDA, Center for Drug Evaluation and Research, Liposome drug products chemistry, manufacturing, and controls; human pharmacokinetics and bioavailability; and labeling documentation guidance for industry (2015)
  73. Jiang Wenlei, Lionberger Robert, Yu Lawrence X, In vitro and in vivo characterizations of PEGylated liposomal doxorubicin, 10.4155/bio.10.204
  74. Tibbitt Mark W., Rodell Christopher B., Burdick Jason A., Anseth Kristi S., Progress in material design for biomedical applications, 10.1073/pnas.1516247112