Pharmaceutical Biotechnology: Fundamentals and Applications PDF
Preview Pharmaceutical Biotechnology: Fundamentals and Applications
Daan J. A. Crommelin Robert D. Sindelar Bernd Meibohm Editors Pharmaceutical Biotechnology Fundamentals and Applications Fifth Edition Pharmaceutical Biotechnology Daan J. A. Crommelin • Robert D. Sindelar Bernd Meibohm Editors Pharmaceutical Biotechnology Fundamentals and Applications Fifth Edition Editors Daan J. A. Crommelin Robert D. Sindelar Utrecht University Faculty of Pharmaceutical Sciences and The Utrecht Centre for Health Evaluation & Outcomes The Netherlands Sciences (CHEOS) The University of British Columbia (UBC) Bernd Meibohm Vancouver, BC College of Pharmacy Canada University of Tennessee Health Science Center Memphis, TN USA ISBN 978-3-030-00709-6 ISBN 978-3-030-00710-2 (eBook) https://doi.org/10.1007/978-3-030-00710-2 © Springer Nature Switzerland AG 2013, 2019 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. The publisher remains neutral with regard to jurisdictional claims in published maps and institutional affiliations. This Springer imprint is published by the registered company Springer Nature Switzerland AG The registered company address is: Gewerbestrasse 11, 6330 Cham, Switzerland Contents 1 Molecular Biotechnology: From DNA Sequence to Therapeutic Protein . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1 Ronald S. Oosting 2 Biophysical and Biochemical Characteristics of Therapeutic Proteins . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19 Wim Jiskoot and Daan J. A. Crommelin 3 Protein Stability and Characterization . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33 Atanas Koulov 4 Production and Purification of Recombinant Proteins . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57 Alfred Luitjens and Emile van Corven 5 Formulation of Biologics Including Biopharmaceutical Considerations . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 83 Daan J. A. Crommelin, Andrea Hawe, and Wim Jiskoot 6 Pharmacokinetics and Pharmacodynamics of Therapeutic Peptides and Proteins . . . . . . . . . . . . . . . . . . . . .105 Bernd Meibohm 7 Immunogenicity of Therapeutic Proteins . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .139 Wim Jiskoot, Theo Rispens, and Grzegorz Kijanka 8 Monoclonal Antibodies: From Structure to Therapeutic Application . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .151 Rong Deng, C. Andrew Boswell, Wendy S. Putnam, Meina T. Tang, Amit Garg, Chunze Li, Shan Chung, and Sandhya Girish 9 Genomics, Other “OMIC” Technologies, Precision Medicine, and Additional Biotechnology-Related Techniques. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .191 Robert D. Sindelar 10 Dispensing Biotechnology Products: Handling, Professional Education, and Product Information . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .239 Robert D. Sindelar 11 Economic Considerations in Medical Biotechnology . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .253 Amit S. Patel and Kartick P. Shirur 12 Regulatory Framework for Biosimilars . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .265 Vinod P. Shah and Daan J. A. Crommelin 13 An Evidence-Based Practice Approach to Evaluating Biotechnologically Derived Medications . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .275 James P. McCormack 14 Vaccines . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .281 Wim Jiskoot, Gideon F. A. Kersten, Enrico Mastrobattista, and Bram Slütter 15 Oligonucleotides . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .305 Raymond M. Schiffelers, Erik Oude Blenke, and Enrico Mastrobattista vi Contents 16 Gene Therapy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .323 Hao Wu, Amit Kumar Chaudhary, and Ram I. Mahato 17 Advanced Therapies: Clinical, Non-clinical and Quality Considerations . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .357 Karin H. Hoogendoorn 18 Insulin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .403 John M. Beals, Michael R. DeFelippis, Chad D. Paavola, David P. Allen, Ashish Garg, and D. Bruce Baldwin 19 Follicle-Stimulating Hormone . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .429 Tom Sam and Renato de Leeuw 20 Human Growth Hormone . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .437 Le N. Dao, Barbara Lippe, Michael Laird, and Ingrid Beierle 21 Recombinant Coagulation Factors and Thrombolytic Agents . . . . . . . . . . . . . . .451 Koen Mertens and Alexander B. Meijer 22 Recombinant Human Deoxyribonuclease I . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .471 Robert A. Lazarus and Jeffrey S. Wagener† 23 Monoclonal Antibodies in Cancer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .489 Jürgen Barth 24 Hematopoietic Growth Factors . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .521 Juan Jose Pérez-Ruixo 25 Monoclonal Antibodies in Solid Organ Transplantation . . . . . . . . . . . . . . . . . . .537 Nicole A. Pilch, Holly B. Meadows, and Rita R. Alloway 26 Antibody-Based Biotherapeutics in Inflammatory Diseases . . . . . . . . . . . . . . . .557 Honghui Zhou, Yan Xu, and Amarnath Sharma 27 Interferons and Interleukins . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .619 Jean-Charles Ryff and Sidney Pestka† Index . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .645 Preface Over the past 30 years, the share of biotechnologically derived drug products in the arsenal of medicinal products has been growing steadily. These drug products include proteins, such as monoclonal antibodies, antibody fragments, endogenous or modified hormones, and growth factors, as well as antisense oligonucleotides, RNA, DNA preparations for gene therapy, and stem cell therapies. In 2017, 12 out of the 46 approved marketing authorizations for new molecular entities by the US Food and Drug Administration (FDA) were biotech products, mainly from the monoclonal antibody family (Mullard 2018). Drug products such as epoetin-α (Epogen®, Eprex®, Procrit®), interferon-α (Intron®A, Roferon®A) and interferon-β (Avonex®, Rebif®, Betaseron®), anti-TNF-α agents, etanercept (Enbrel®), infliximab (Remicade®), adali- mumab (Humira®), bevacizumab (Avastin®), and trastuzumab (Herceptin®) are all examples of highly successful biotech drugs that have revolutionized the pharmaco- therapy of previously unmet medical needs. And, last but not least, biotech drugs also have a major socioeconomic impact. In 2017, 7 of the 10 top selling drugs in the world were biotechnologically derived drug products, with sales varying between 5–16 billion US dollars, totaling 75 billion dollars. The revenues of biotechnology- based medication are annually growing at a 10% pace and will reach 300 billion US dollars in 2021, i.e., one third of the total global revenues for brand medicines in that year (IFPIA 2017). The techniques of biotechnology are a driving force of modern drug discovery as well. Due to the rapid growth in the importance of biopharmaceuticals and the techniques of biotechnologies to modern medicine and the life sciences, the field of pharmaceutical biotechnology has become an increasingly important component in the education of today’s and tomorrow’s pharmacists and pharmaceutical scientists. We believe that there is a critical need for an introductory textbook on Pharmaceutical Biotechnology that provides well-integrated, detailed coverage of both the relevant science and clinical application of pharmaceuticals derived by biotechnology. Previous editions of the textbook Pharmaceutical Biotechnology: Fundamentals and Applications have provided a well-balanced framework for education in various aspects of pharmaceutical biotechnology, including production, dosage forms, admin- istration, economic and regulatory aspects, and therapeutic applications. Rapid growth and advances in the field of pharmaceutical biotechnology, however, made it necessary to revise this textbook in order to provide up- to- date information and intro- duce readers to the cutting-edge knowledge and technology of this field. This fifth edition of the textbook Pharmaceutical Biotechnology: Fundamentals and Applications builds on the successful concept used in the preceding editions and fur- ther expands its availability as electronic versions of the full book as well as individ- ual chapters are readily available and downloadable through online platforms. The textbook is structured into two sections. An initial basic science and general features section comprises the first 17 chapters introducing the reader to key concepts at the foundation of the technology relevant for protein therapeutics, including molecular biology, production and analytical procedures, formulation development, pharmacokinetics and pharmacodynamics, immunogenicity, and chapters dealing viii PrefaCe with regulatory, economic, and pharmacy practice considerations and with evolving new technologies and applications. The second section (Chaps. 18–27) discusses the various therapeutic classes of protein biologics and nucleotide- and cell-b ased therapeutics. All chapters of the previous edition were revised; some were completely over- hauled and two added (“Analytical Toolbox” and “Therapeutic Proteins in Evidence- Based Practice”). The section on monoclonal antibodies is differentiated into a section on general considerations for this important class of biologics and sections focused on their application in oncology, transplantation, and inflammation in order to allow for a comprehensive discussion of the substantial number of approved antibody drugs (see above). In accordance with previous editions, the new edition of Pharmaceutical Biotechnology: Fundamentals and Applications will have as a primary target students in undergraduate and professional pharmacy programs as well as graduate students in the pharmaceutical sciences. Additional important audiences are pharmaceutical sci- entists in industry and academia, particularly those that have not received formal training in pharmaceutical biotechnology and are inexperienced in this field. We are convinced that this fifth edition of Pharmaceutical Biotechnology: Fundamentals and Applications makes an important contribution to the education of pharmaceutical scientists, pharmacists, and other healthcare professionals as well as serving as a ready resource on biotechnology. By increasing the knowledge and exper- tise in the development, application, and therapeutic use of “biotech” drugs, we hope to help facilitate a widespread, rational, and safe application of this important and rapidly evolving class of therapeutics. Utrecht, The Netherlands Daan J. A. Crommelin Vancouver, BC, Canada Robert D. Sindelar Memphis, TN, USA Bernd Meibohm March 2019 REFERENCES IFPIA (2017) The pharmaceutical industry and global health: facts and figures 2017. https:// www.ifpma.org/wp-content/uploads/2017/02/IFPMA-Facts-And-Figures-2017.pdf Mullard A (2018) FDA drug approvals 2017. Nat Rev Drug Discov 17:81–85 Abbreviations α2-PI α2-Plasmin inhibitor γ-RV Gamma retrovirus μg Micrograms 3D Three-dimensional 4-OHT 4-hydroxytamoxifen 5-FC 5-Fluorocytosine 5-FU 5-Fluorouridine AAV Adeno associated virus AAV2 AAV serotype 2 Ad Adenovirus ADA Adenosine deaminase deficiency ADA Anti-drug antibodies ADA Adenosine deaminase ADCC Antibody-dependent cellular cytotoxicity ADME Absorption, distribution, metabolism, elimination ADRs Adverse drug reactions AEX Anion exchange AF4 Asymmetrical flow field-flow fractionation AHFS American Hospital Formulary Service AI Artificial Intelligence AIDS Acquired immunodeficiency syndrome aie After initial entry into vial ALL Acute lymphoblastic leukaemia AMD Age related macular degeneration APC Activated protein C APC Antigen-presenting cell APE1 Apurinic/apyrimidinic endonucleases from human API Active pharmaceutical ingredient ART Assisted reproductive technologies ASO Antisense oligonucleotide ATF Alternating tangential flow ATMPs Advanced therapy medicinal products AUC Area under the curve/analytical ultracentrifugation BCA Bicinchoninic Acid BCG Bacillus Calmette-Guérin BCGF B-cell growth factor BCR B-cell receptor BDD-FVIII B-domain deleted factor VIII BFU Bone marrow erythroid progenitor cells BHK Baby hamster kidney BM Bone marrow BMP Bone morphogenetic protein BPCI Biologics Price Competition and Innovation BRMs Biological (immune) response modifiers x abbreviations BSA Bovine serum albumin BSE Bovine spongiform encephalopathy BSE/TSE Bovine/transmittable spongiform encephalopathy, mad cow disease BSI British standards institution CAPEX Capital expenditures CAR Chimeric antigen receptor CAR Coxsackievirus and adenovirus receptors Cas CRISPR-associated proteins Cas9 CRISPR-associated protein 9 CAT Committee for advanced therapies CBA Cost-benefit analysis CBER Center for Biologics Evaluation and Research CD Circular dichroism spectroscopy CD Cluster of differentiation CD Crohn’s disease CD Cytosine deaminase cDNA Copy or Complementary DNA CDR Complementarity-determining region CE SDS Capillary electrophoresis sodium dodecyl sulfate CEA Carcinoembryonic antigen CEA Cost-effectiveness analysis CEX Cation exchange CF Cystic fibrosis CFR Code of Federal Regulations CFTR Cystic fibrosis transmembrane conductance regulator CG Chorionic gonadotropin CGH Comparative genomic hybridization cGMP Current Good Manufacturing Practice CHMP Committee for Medicinal Products for Human Use CHO cells Chinese hamster ovary cells CHO Chinese hamster ovary CIP Clean-in-place CK Chemokines CLL Chronic lymphocytic leukemia CM Capacitance manometer CMA Cost-minimization analysis CMA Critical material attribute CMC Chemical, manufacturing and controls CMS US Centers for Medicare & Medicaid Services CMV Cytomegalovirus COI Cost of illness COPD Chronic obstructive pulmonary disease COS Controlled ovarian stimulation CpG Cytosine-phosphodiester-guanine CPG2 Carboxypeptidase G2 CPP Cell-penetrating peptide CPP Critical process parameters CQA Critical quality attribute CRISPR Cluster regularly interspaced short palindrome repeat CSF Cerebrospinal fluid CSF-1R Colony-stimulating factor 1 receptor CT Cholera toxin CTI Computed tomography imaging CTL Cytotoxic T lymphocyte CTP Carboxy terminal peptide
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