AAPS Advances in the Pharmaceutical Sciences Series

Transcription

1 AAPS Advances in the Pharmaceutical Sciences Series Volume 20 Series Editors Daan J. A. Crommelin Utrecht University, Utrecht, The Netherlands Robert A. Lipper Back Cove Pharma, LLC, Waldoboro, Maine, USA

2 The AAPS Advances in the Pharmaceutical Sciences Series, published in partnership with the American Association of Pharmaceutical Scientists, is designed to deliver well written volumes authored by opinion leaders and authorities from around the globe, addressing innovations in drug research and development, and best practice for scientists and industry professionals in the pharma and biotech industries. For more details and to see a list of titles in the Series please visit springer.com/series/8825

3 Daan J.A. Crommelin Jon S. B. de Vlieger Editors Non-Biological Complex Drugs The Science and the Regulatory Landscape 1 3

4 Editors Daan J.A. Crommelin Utrecht University Utrecht The Netherlands Jon S. B. de Vlieger Top Institute Pharma NBCD Working Group Leiden The Netherlands ISSN ISSN X (Electronic) AAPS Advances in the Pharmaceutical Sciences Series ISBN ISBN (ebook) DOI / Library of Congress Control Number: Springer Cham Heidelberg New York Dordrecht London Springer International Publishing Switzerland 2015 This work is subject to copyright. All rights are reserved by the Publisher, whether the whole or part of the material is concerned, specifically the rights of translation, reprinting, reuse of illustrations, recitation, broadcasting, reproduction on microfilms or in any other physical way, and transmission or information storage and retrieval, electronic adaptation, computer software, or by similar or dissimilar methodology now known or hereafter developed. The use of general descriptive names, registered names, trademarks, service marks, etc. in this publication does not imply, even in the absence of a specific statement, that such names are exempt from the relevant protective laws and regulations and therefore free for general use. The publisher, the authors and the editors are safe to assume that the advice and information in this book are believed to be true and accurate at the date of publication. Neither the publisher nor the authors or the editors give a warranty, express or implied, with respect to the material contained herein or for any errors or omissions that may have been made. Printed on acid-free paper Springer is part of Springer Science+Business Media (

5 Contents Introduction: Defining the Position of Non-Biological Complex Drugs... 1 Daan J. A. Crommelin, Jon S. B. de Vlieger and Stefan Mühlebach Part I Non Biological Complex Drugs Polymeric Micelles Ethlinn V.B. van Gaal and Daan J.A. Crommelin Liposomes: The Science and the Regulatory Landscape Daan J.A. Crommelin, Josbert M. Metselaar and Gert Storm Glatiramoids Vera Weinstein, Rivka Schwartz, Iris Grossman, Benjamin Zeskind and J. Michael Nicholas Iron Carbohydrate Complexes: Characteristics and Regulatory Challenges Stefan Mühlebach and Beat Flühmann Drug Nanocrystals Gerrit Borchard Part II Characterization of NBCDs; Analytical Tools to Consider Analytical Methods for Determining the Size (Distribution) in Parenteral Dispersions David F. Driscoll and David F. Nicoli NBCD Pharmacokinetics and Bioanalytical Methods to Measure Drug Release Vishakha V. Ambardekar and Stephan T. Stern v

6 vi Contents Part III Closely related Complex Drugs Low Molecular Weight Heparins, Biological Drugs close to Non-Biological Complex Drugs Isabel Rodrigo, Sofía Caruncho, Concepción Alonso, Antonio Gómez-Outes and Barbara Mulloy Nanoparticle Albumin-Bound Anticancer Agents Neil Desai Part IV Regulatory landscape and outlook The EU Regulatory Landscape of Non-Biological Complex Drugs Ruben Pita Epilogue: What Did We Learn? What Can We Expect in the Future? Concluding Remarks and Outstanding Issues Daan J. A. Crommelin and Jon S. B. de Vlieger Index

7 Contributors Concepción Alonso Biological Products and Biotechnology Division, Medicines for Human Use Department, Spanish Agency for Medicines and Medical Devices (AEMPS), Madrid, Spain Vishakha V. Ambardekar Nanotechnology Characterization Laboratory, Cancer Research Technology Program, Leidos Biomedical Research, Inc., Frederick National Laboratory for Cancer Research, Frederick, MD, USA Gerrit Borchard School of Pharmaceutical Sciences Geneva-Lausanne, University of Geneva, University of Lausanne, Geneva, Switzerland Sofía Caruncho Biological Products and Biotechnology Division, Medicines for Human Use Department, Spanish Agency for Medicines and Medical Devices (AEMPS), Madrid, Spain Daan J.A. Crommelin Department Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands Department Pharmaceutics, Utrecht Institute for Pharmaceutical Sciences, UIPS, Utrecht University, Utrecht, The Netherlands Department of Pharmaceutics, Utrecht University, Utrecht, The Netherlands Jon S. B. de Vlieger NBCD Working Group, Top Institute Pharma, Leiden, The Netherlands Neil Desai Strategic Platforms, Celgene Corporation (Abraxis BioScience), Los Angeles, CA, USA David F. Driscoll Stable Solutions LLC, Easton, MA, USA UMASS Medical School, Worcester, MA, USA Beat Flühmann Department of Global Regulatory Affairs, Vifor Pharma Ltd., Glattbrugg, Switzerland vii

8 viii Contributors Antonio Gómez-Outes Pharmacology and Clinical Evaluation Division, Medicines for Human Use Department, Spanish Agency for Medicines and Medical Devices (AEMPS), Madrid, Spain Iris Grossman Teva Pharmaceutical Industries, Ltd., Petach Tikva, Israel Stefan Mühlebach Department of Global Regulatory Affairs, Vifor Pharma Ltd., Glattbrugg, Switzerland Department of Pharmaceutical Sciences Division of Clinical Pharmacy & Epidemiology, University of Basel, Basel, Switzerland Josbert M. Metselaar Enceladus Pharmaceuticals BV, Naarden, The Netherlands Barbara Mulloy Institute of Pharmaceutical Science, King s College London, London, UK J. Michael Nicholas Teva Pharmaceuticals, Kansas City, MO, USA David F. Nicoli Stable Solutions LLC, Goleta, CA, USA Particle Sizing Systems LLC, Port Richey, FL, USA Ruben Pita European Medicine Agency, EMA, London, UK Isabel Rodrigo Biological Products and Biotechnology Division, Medicines for Human Use Department, Spanish Agency for Medicines and Medical Devices (AEMPS), Madrid, Spain Rivka Schwartz Teva Pharmaceutical Industries, Ltd., Petach Tikva, Israel Stephan T. Stern Nanotechnology Characterization Laboratory, Cancer Research Technology Program, Leidos Biomedical Research, Inc., Frederick National Laboratory for Cancer Research, Frederick, MD, USA Gert Storm Department Pharmaceutics, Utrecht Institute for Pharmaceutical Sciences, UIPS, Utrecht University, Utrecht, The Netherlands Ethlinn V.B. van Gaal OctoPlus B.V., Dr. Reddy s Laboratories Ltd., Leiden, The Netherlands Vera Weinstein Teva Pharmaceutical Industries, Ltd., Petach Tikva, Israel Benjamin Zeskind Immuneering Corporation, Cambridge, USA

9 About the Editors Prof. Daan Crommelin is emeritus-professor at the Department of Pharmaceutics at Utrecht University. Until December 2011 he was scientific director of the Dutch Top Institute Pharma in Leiden. He is adjunct professor at the Department of Pharmaceutics and Pharmaceutical Chemistry at the University of Utah. Crommelin is co-founder of OctoPlus, a Leiden based company specialized in the development of pharmaceutical (mainly protein based) product formulations and advanced drug delivery systems. He published extensively and is on the editorial board of 10+ peer reviewed journals in the pharmaceutical sciences. Dr. Jon S. B. de Vlieger obtained his doctoral degree in bio analytical chemistry from the VU University in Amsterdam. He is currently involved in the strategy department of the Dutch Top Institute Pharma, a not-for-profit organization catalyzing the development of medicines by establishing partnerships and actively managing research programs. For TI Pharma he coordinates several international public private partnerships, including the Non Biological Complex Drugs Working Group, an international network of scientific and clinical experts from academia, industry and regulatory bodies, with expertise in many aspects of the development and evaluation of NBCDs. ix