4. Nonclinical Safety Assessments and Clinical Pharmacokinetics for Oligonucleotide Therapeutics: A Regulatory Perspective

  1. Kun Cheng3 and
  2. Ram I. Mahato4
  1. Shwu-Luan Lee1,
  2. Paul Brown1,
  3. Jian Wang2 and
  4. Robert T. Dorsam1

Published Online: 26 APR 2013

DOI: 10.1002/9781118610749.ch4

Advanced Delivery and Therapeutic Applications of RNAi

Advanced Delivery and Therapeutic Applications of RNAi

How to Cite

Lee, S.-L., Brown, P., Wang, J. and Dorsam, R. T. (2013) Nonclinical Safety Assessments and Clinical Pharmacokinetics for Oligonucleotide Therapeutics: A Regulatory Perspective, in Advanced Delivery and Therapeutic Applications of RNAi (eds K. Cheng and R. I. Mahato), John Wiley and Sons, Ltd, Chichester, UK. doi: 10.1002/9781118610749.ch4

Editor Information

  1. 3

    School of Pharmacy, University of Missouri-Kansas City, USA

  2. 4

    Department of Pharmaceutical Sciences, University of Tennessee Health Science Center, USA

Author Information

  1. 1

    Office of New Drugs, Center for Drug Evaluation and Research, US Food and Drug Administration, USA

  2. 2

    Office of Translational Sciences, Center for Drug Evaluation and Research, US Food and Drug Administration, USA

Publication History

  1. Published Online: 26 APR 2013
  2. Published Print: 10 JUN 2013

ISBN Information

Print ISBN: 9781119976868

Online ISBN: 9781118610749

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Keywords:

  • International Conference on Harmonization (ICH) guidance;
  • regulatory experience;
  • pharmacology/toxicology;
  • clinical pharmacology;
  • investigational new drug (IND);
  • new drug application (NDA)

Summary

Oligonucleotide-based therapeutics (ODN) are regulated by the US FDA as small-molecule drug products in terms of review process, ICH guidance, and general studies requirements in regulatory submissions. This chapter provides pharmacology/toxicology and clinical pharmacology regulatory perspectives on ODNs and identifies challenges that are somewhat unique to this class of therapeutics.