Nanomedicines: Addressing the scientific and regulatory gap

Annals of the New York Academy of Sciences

Volumn 1313, Issue 1, 2014, Pages 35-56

  Tinkle, Sally ^a   Mcneil, Scott E ^b   Mühlebach, Stefan ^c,d   Bawa, Raj ^e,f   Borchard, Gerrit ^g   Barenholz, Yechezkel Chezy ^h   Tamarkin, Lawrence ⁱ   Desai, Neil ^j  

^a Science and Technology Policy Institute   (United States)

^b FREDERICK NATIONAL LABORATORY FOR CANCER RESEARCH   (United States)

^c Vifor (International) Inc   (Switzerland)

^d UNIVERSITY OF BASEL   (Switzerland)

^e Bawa Biotechnology Consulting LLC   (United States)

^f RENSSELAER POLYTECHNIC INSTITUTE   (United States)

^g UNIVERSITY OF GENEVA   (Switzerland)

^h HEBREW UNIVERSITY   (Israel)

ⁱ CytImmune Sciences Inc   (United States)

^j Abraxis Bioscience   (United States)

more..

Author keywords

Abbreviated new drug application (ANDA); Bioequivalence; Biosimilars; Follow on biologics; Immunogenicity; Nanomedicine; Nanopharmaceuticals; Nanosimilars; Nanotechnology; non biologic complex drug (NBCD); Patents; Pharmacodynamics; Pharmacokinetics; Regulation

Indexed keywords

AMPHOTERICIN B CHOLESTEROL SULFATE; AMPHOTERICIN B LIPID COMPLEX; APREPITANT; ASPARAGINASE MACROGOL; BIOSIMILAR AGENT; CARBON NANOTUBE; CYTARABINE; DAUNORUBICIN; DOXORUBICIN; ESTRADIOL; FENOFIBRATE; GLATIRAMER; GOLD NANOPARTICLE; HEPATITIS A VACCINE; NANOMATERIAL; NANOSIMILAR AGENT; PACLITAXEL; PEGADEMASE; PEGAPTANIB; PEGINTERFERON ALPHA2A; PEGINTERFERON ALPHA2B; PEGVISOMANT; PROPOFOL; RAPAMYCIN; RECOMBINANT GRANULOCYTE COLONY STIMULATING FACTOR; SEVELAMER; UNCLASSIFIED DRUG;

ARTICLE; BIOEQUIVALENCE; DISEASE CONTROL; DRUG APPROVAL; DRUG CONTROL; DRUG DELIVERY SYSTEM; DRUG EFFICACY; FOOD AND DRUG ADMINISTRATION; HUMAN; INVENTION; MALIGNANT NEOPLASTIC DISEASE; MEDICAL RESEARCH; MICELLE; NANOMEDICINE; NANOTECHNOLOGY; PATENT; PATIENT CARE; PHARMACEUTICAL EQUIVALENCE; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PRODUCT DEVELOPMENT; PRODUCT SAFETY; PROPHYLAXIS; RANDOMIZED CONTROLLED TRIAL (TOPIC); UNITED STATES;

ABBREVIATED NEW DRUG APPLICATION (ANDA); BIOEQUIVALENCE; BIOSIMILARS; FOLLOW-ON BIOLOGICS; IMMUNOGENICITY; NANOMEDICINE; NANOPHARMACEUTICALS; NANOSIMILARS; NANOTECHNOLOGY; NON-BIOLOGIC COMPLEX DRUG (NBCD); PATENTS; PHARMACODYNAMICS; PHARMACOKINETICS; REGULATION;

DRUG DELIVERY SYSTEMS; HUMANS; NANOMEDICINE; NANOSTRUCTURES; NANOTECHNOLOGY; RESEARCH;

EID: 84898788471     PISSN: 00778923     EISSN: 17496632     Source Type: Book Series    
DOI: 10.1111/nyas.12403     Document Type: Article

References (54)

1. Ehmann, F., K. Sakai-Kato, R. Duncan, et al. 2013. Next-generation nanomedicines and nanosimlars: EU regulators' initiatives relating to the development and evaluation of nanomedicines. Nanomedicine 8: 849-856
2. Schellekens, H., S. Sven Stegemann, V. Weinstein, et al. 2014. How to regulate NBCD and their follow-on versions: Points to consider. AAPS J 16: 15-21.
3. Borchard, G., B. Flühmann & S. Mühlebach. 2012. Nanoparticle iron medicinal products - Requirements for approval of intended copies of non-biological complex drugs (NBCD) and the importance of clinical comparative studies. Reg Tox Pharmacol 64: 324-328
4. Schellekens, H., E. Klinger, S. Mühlebach, et al. 2011. The therapeutic equivalence of complex drugs. Reg Tox Pharmacol 59: 176-183
5. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2013/09/WC500149496.pdf.
6. Food and Drug Administration [Docket No. FDA-2007-D-0369]. Draft Guidance for Industry on Bioequivalence Recommendations for Iron Sucrose. Published 11/06/2013. Available online at: http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm297630.pdf
7. Mühlebach, S., A. Vulto, J.S.B. de Vlieger, et al. 2013. The authorization of non-biological complex drugs (NBCDs) follow-on versions: specific regulatory and interchangeability rules ahead? GaBi J 2: 204-207.
8. Crommelin, D.J.A., J.S.B. de Vlieger, V. Weinstein, et al. 2014. Different pharmaceutical products need similar terminology. AAPS J 16: 11-14.
9. Hamburg, M.A. 2012. FDA's Approach to Regulation of Nanotechnology Products. Science 336: 299-300. Available online at: http://www.fda.gov/ScienceResearch/SpecialTopics/Nanotechnology/ucm301114.
10. FDA's Nanotechnology Regulatory Science Research Plan (Available online at: http://www.fda.gov/ScienceResearch/SpecialTopics/Nanotechnology/ucm273325.htm)
11. Draft Guidance for Industry: Considering Whether an FDA-Regulated Product Involves the Application of Nanotechnology, June 2011. Available online at: http://www.fda.gov/RegulatoryInformation/Guidances/ucm257698.htm
12. Draft Guidance for Industry: Safety of Nanomaterials in Cosmetic Products, April 2012. Available online at: http://www.fda.gov/Cosmetics/GuidanceComplianceRegulatoryInformation/GuidanceDocuments/ucm300886.htm
13. Draft Guidance for Industry: Assessing the Effects of Significant Manufacturing Process Changes, Including Emerging Technologies, on the Safety and Regulatory Status of Food Ingredients and Food Contact Substances, Including Food Ingredients that are Color Additives, April 2012. Available online at: http://www.fda.gov/Food/GuidanceRegulation/GuidanceDocumentsRegulatoryInformation/IngredientsAdditivesGRASPackaging/ucm300661.htm
14. Overview of CDER Experience with Nanotechnology-related Drugs. Advisory Committee for Pharmaceutical Science and Clinical Pharmacology, August 2012. Available online at: http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/AdvisoryCommitteeforPharmaceuticalScienceandClinicalPharmacology/UCM315773.pdf.
15. FDA/CDER Manual of Policies and Procedures: Reporting Format for Nanotechnology-Related Information in CMC Review, June 2010. Available online at: http://www.fda.gov/downloads/AboutFDA/CentersOffices/CDER/ManualofPoliciesProcedures/UCM214304.pdf
16. FDA/CVM Program Policy and Procedures Manual: Review of ONADE-Regulated Products that Contain Nanomaterials or Otherwise Involve the Use of Nanotechnology, August 2011. Available online at: http://www.fda.gov/downloads/AnimalVeterinary/GuidanceComplianceEnforcement/PoliciesProceduresManual/UCM270271.pdf
17. Mansour, H.M., C-W. Park & R. Bawa. 2014. Design and development of approved nanopharmaceutical products. In Handbook of Clinical Nanomedicine: From Bench to Bedside. R. Bawa, G. Audette & I. Rubinstein, Eds. Singapore: Pan Stanford Publishing. Available May 31, 2014.
18. Bawa, R. 2008. Nanoparticle-based therapeutics in humans: a survey. Nanotechnology Law & Business 5: 135-155.
19. Bawa, R., S. Melethil, W. J. Simmons & D. Harris. 2008. Nanopharmaceuticals - patenting issues and FDA regulatory challenges. American Bar Association SciTech Lawyer 5: 10-15.
20. Bawa, R. 2013. FDA and nanotech: Baby steps lead to regulatory uncertainty. In Bionanotechnology: A revolution in biomedical sciences and human health. D. Bagchi, et al., Eds: 720-732. UK: Wiley Blackwell.
21. Johnson, D. 2014. [Posted 26 Nov 2013 on Nanoclast, IEEE Spectrum's nanotechnology blog] Nanomedicines will be big once they emerge from regulatory troubles. Available online at: http://spectrum.ieee.org/nanoclast/at-work/innovation/nanomedicines-will-be-big-once-they-emerge-from-regulatory-troubles
22. Harris, D. & R. Bawa. 2007. The carbon nanotube patent landscape in nanomedicine: an expert opinion. Expert Opinion on Therapeutic Patents 17: 1165-1174.
23. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM199635.pdf
24. https://www.fbo.gov/?s=opportunity&mode=form&id=14193764562d3ee694924265d4fc3356&tab=core&_cview=0
25. http://www.edqm.eu/en/european-pharmacopoeia-membership-observership-608.html
26. http://www.edqm.eu/en/european-pharmacopoeia-background-50.html
27. Barenholz, Y. 2012. Doxil®-The First FDA-Approved Nano-Drug: Lessons Learned. J. Cont. Rel. 160: 117-134.
28. Solomon, R. & A. Gabizon. 2008. Clinical pharmacology of liposomal anthracyclines: Focus on pegylated liposomal doxorubicin. Clinical Lymphoma Myeloma 8: 21-32.
29. Ferrandina, G., A. Licameli, D. Lorusso, et al. 2010. Pegylated liposomal doxorubicin in the management of ovarian cancer. Therapeutics and Clinical Risk Management. 6: 463-483.
30. Gabizon, A., H. Shmeeda & Y. Barenholz. 2003. Pharmacokinetics of pegylated liposomal Doxorubicin: Review of animal and human studies. Clin. Pharmacokinetics. 42: 419-436.
31. Gabizon, A., R. Catane, B. Uziely, et al. 1994. Prolonged circulation time and enhanced accumulation in malignant exudates of doxorubicin encapsulated in polyethylene-glycol coated liposomes. Cancer Res. 54: 987-992.
32. Haran, G., R. Cohen, L.K. Bar & Y. Barenholz. 1993. Transmembrane ammonium sulfate gradients in liposomes produce efficient and stable entrapment of amphipathic weak bases. Biochim. Biophys. Acta 1151: 201-215.
33. Barenholz, Y. 2001. Liposome application: problems and prospects. Curr. Opin. Colloid Interface Sci. 6: 66-77.
34. Barenholz, Y. 2007. Amphipathic weak base loading into preformed liposomes having a transmembrane ammonium ion gradient: From the bench to approved DOXIL. In Liposome Technology, 3rd Edition. Vol. 2. G. Gregoriadis, Ed.: 1-25. New York: Informa Healthcare.
35. Jain, R.K. 1987. Transport of molecules in the tumor interstitium: a review. Cancer Res 47: 3039-3051.
36. Maeda, H., G.Y. Bharate & J. Daruwalla. 2009. Polymeric drugs for efficient tumor-targeted drug delivery based on EPR-effect. Eur J Pharm Biopharm 71: 409-419.
37. Barenholz, Y. 2011. Doxil®-The First FDA-Approved Nano-Drug: From an Idea to a Product. In Harnessing Biomaterials for Nanomedicine: Preparation, Toxicity and Applications. D. Peer, Ed.: 335-398. Pan Stanford Publishing Singapore.
38. Gabizon, A., R. Chisin, S. Amselem, et al. 1991. Pharmacokinetic and imaging studies in patients receiving a formulation of liposome-associated Adriamycin. Br. J. Cancer 64: 1125-1132.
39. Barenholz, Y. & G. Haran. 1994. "Liposomes: Efficient Loading and Controlled Release of Amphipathic Molecules". U.S. Patent 5, 316, 771, issued on May 31, 1994.
40. Jiang, W., R. Lionberger & L.X. Yu. 2011. In vitro and in vivo characterizations of PEGylated liposomal doxorubicin. Bioanalysis 3: 333-344.
41. Szebeni, J., F. Muggia, A. Gabizon & Y. Barenholz. 2011. Activation of complement by therapeutic liposomes and other lipid excipient-based therapeutic products: Prediction and prevention. Advanced Drug Delivery Reviews 63: 1020-1030.
42. Szebeni, J., P. Bedocs, Z. Rozsynyay, et al. 2012. Liposome-induced complement activation and related cardiopulmonary distress in pigs: factors promoting reactogenity of Doxil and AmBisome. Nanomedicine 8: 176-184.
43. Barenholz, Y. & S. Amselem. 1993. Quality control assays in the development and clinical use of liposome based formulations. In Liposome Technology, 2nd Edition, Vol. I, Liposome Preparation and Related Techniques. G. Gregoriadis, Ed.: 527-616. Boca Raton, FL: CRC Press.
44. Lasic, D.D., P.M. Frederik, M.C.A. Stuart, et al. 1992. Gelation of liposome interior. A novel method for drug encapsulation. FEBS Lett. 312: 255-258.
45. Zucker, D., A.V. Andriyanov, A. Steiner, et al. 2012. Characterization of PEGylated nanoliposomes co-remotely loaded with topotecan and vincristine: relating structure and pharmacokinetics to therapy efficacy. J. Cont. Rel. 160: 281-289.
46. Fukumura, D & R.K. Jain. 2007. Tumor microenvironment abnormalities: causes, consequences, and strategies to normalize. Journal of Cellular Biochemistry 101: 937-949.
47. Paciotti, G.F., L. Myer, D. Weinreich, et al. 2004. Colloidal gold: a novel nanoparticle vector for tumor directed drug delivery. Drug Delivery 11: 169-183.
48. Farma, J.M., M. Puhlmann, P.A. Soriano, et al. 2007. Direct evidence for rapid and selective induction of tumor neovascular permeability by tumor necrosis factor and a novel derivative, colloidal gold bound tumor necrosis factor. Int. J. Cancer 120: 2474-2480.
49. van Etten, B., A.N. van Geel, J.H.W. de Wilt & A.M.M. Eggermont. 2003. Fifty tumor necrosis factor-based isolated limb perfusions for limb salvage in patients older than 75 years with limb- threatening soft tissue sarcomas and other extremity tumors. Annals of Surgical Oncology 10: 32-37.
50. Libutti, S.K., G.F. Paciotti, A.A. Byrnes, et al. 2010. Phase I and pharmacokinetic studies of CYT-6091, a novel PEGylated colloidal gold-rhTNF nanomedicine. Clin Cancer Res 16: 6139-6149.
51. Hawkins, M.J., P. Soon-Shiong & N. Desai. 2008. Protein nanoparticles as drug carriers in clinical medicine. Adv Drug Deliv Rev 60: 876-885.
52. Irizarry, L., T. Luu, J. McKoy, et al. 2009. Cremophor EL-containing paclitaxel-induced anaphylaxis: a call to action. Community Oncology 6: 132-134.
53. FDA: Draft Guidance on Paclitaxel, 2012. Available online at: http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm320015.pdf
54. Bawa, R. 2009. Patenting inventions in bionanotechnology - A primer for scientists and lawyers. In Bionanotechnology: Global Prospects. D.E. Reisner, Ed.: 309-337. Boca Raton, Florida: CRC Press.