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Handbook of Biological
Therapeutic Proteins
Since 1972, which marks the invention of recombinant engineering, more than 500 therapeutic
proteins have been approved for clinical use. Today, biological drugs constitute almost 70% of all
new drugs and have a biological origin. The rst edition of this book dealt with biosimilars, and
this edition (i.e., the second edition) focuses on new drugs, yet limits to therapeutic proteins. Newer
technologies for drug development represent the updated topics in the book and include repurposing, AI- driven identication of newer designs, novel expression systems, manufacturing using
these systems, rapidly changing regulatory pathways, and legal hurdles. This edition discusses how
to identify, develop, manufacture, and take multibillion dollar products to market within the shortest
possible time.
Features:
• Complete and thorough coverage of the regulatory and technological challenges of developing
generic therapeutic proteins
• Comprehensive analysis, discovery to market, newer technologies, regulatory planning and
intellectual property– related hurdles are included, and this information is not found elsewhere
• Expanded volume that must be in the hands of every company interested in biological drugs,
including mRNA- based biopharmaceutical companies that quickly enter into the market
• Discusses how to identify, develop, manufacture, and take multibillion dollar products to
market within the shortest possible time
• Renowned author and entrepreneur in the eld of drug discovery and production
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Handbook of Biological
Therapeutic Proteins
Regulatory, Manufacturing, Testing,
and Patent Issues
Second Edition
Authored by
Sarfaraz K. Niazi
Adjunct Professor at the University of Illinois
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Second edition published 2024
by CRC Press
2385 Executive Center Drive, Suite 320, Boca Raton, FL 33431
and by CRC Press
4 Park Square, Milton Park, Abingdon, Oxon, OX14 4RN
© 2024 Sarfaraz K. Niazi
CRC Press is an imprint of Taylor & Francis Group, LLC
Reasonable efforts have been made to publish reliable data and information, but the author and publisher cannot assume
responsibility for the validity of all materials or the consequences of their use. The authors and publishers have attempted to
trace the copyright holders of all material reproduced in this publication and apologize to copyright holders if permission to
publish in this form has not been obtained. If any copyright material has not been acknowledged please write and let us know
so we may rectify in any future reprint.
Except as permitted under U.S. Copyright Law, no part of this book may be reprinted, reproduced, transmitted, or utilized
in any form by any electronic, mechanical, or other means, now known or hereafter invented, including photocopying,
microlming, and recording, or in any information storage or retrieval system, without written permission from the publishers.
For permission to photocopy or use material electronically from this work, access www.copyri ght.com or contact the
Copyright Clearance Center, Inc. (CCC), 222 Rosewood Drive, Danvers, MA 01923, 978- 750- 8400. For works that are not
available on CCC please contact mpkbookspermissions@tandf.co.uk
Trademark notice: Product or corporate names may be trademarks or registered trademarks and are used only for identication
and explanation without intent to infringe.
Library of Congress Cataloging‑in‑Publication Data
Names: Niazi, Sarfaraz, 1949– author.
Title: Handbook of biological therapeutic proteins : regulatory, manufacturing, testing, and patent issues / authored by
Sarfaraz K. Niazi.
Other titles: Handbook of biogeneric therapeutic proteins Description: Second edition | Boca Raton : CRC Press, 2024. |
Preceded by Handbook of biogeneric therapeutic proteins / Sarfaraz K. Niazi. 2006. | Includes bibliographical references
and index. | Summary: “Since 1972 when recombinant engineering was invented, over 500 therapeutic proteins have been
approved. Today, biological drugs constitute almost 70% of all new drugs and are of biological origin. The rst edition of
this book dealt with biosimilars, the second focuses on new drugs yet limits to therapeutic proteins. The newer technologies
for development represent the updated topics in the book and include repurposing, AI-driven identication of newer designs,
novel expression systems and manufacturing, fast changing regulatory pathways, and legal hurdles. Discusses how to identify,
develop, manufacture and take multibillion dollar products to market in the shortest possible time”– Provided by publisher.
Identiers: LCCN 2023044890 (print) | LCCN 2023044891 (ebook) | ISBN 9781032489605 (hardback) |
ISBN 9781032490540 (paperback) | ISBN 9781003392026 (ebook)
Subjects: MESH: Recombinant Proteins–therapeutic use | Biosimilar Pharmaceuticals | Technology, Pharmaceutical |
Drug Industry
Classication: LCC RM300 (print) | LCC RM300 (ebook) | NLM QU 55 | DDC 615.1–dc23/eng/20240108
LC record available at https://lccn.loc.gov/2023044890
LC ebook record available at https://lccn.loc.gov/2023044891
ISBN: 9781032489605 (hbk)
ISBN: 9781032490540 (pbk)
ISBN: 9781003392026 (ebk)
DOI: 10.1201/ 9781003392026
Typeset in Times
by Newgen Publishing UK
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v
Contents
Preface ............................................................................................................................................. vii
Author Biography ............................................................................................................................ ix
Introduction by Professor Francisco Baralle .................................................................................... xi
Chapter1 Overview of the Development of Biosimilar Biopharmaceuticals ...............................1
Chapter2 Regulatory Requirements for a Proposed Biosimilar Product ...................................13
Chapter3 Development of a Master Plan for the Biosimilar ......................................................33
Chapter4 Trends in the Manufacturing of Recombinant Proteins .............................................43
Chapter5 Analytical Assessment of a Biosimilar .......................................................................82
Chapter6 Clinical Pharmacology Assessment of a Proposed Biosimilar .................................111
Chapter7 Clinical Immunogenicity Assessment of the Biosimilar ..........................................125
Chapter8 Clinical Efcacy Assessment of the Proposed Biosimilar .......................................149
Chapter9 Recombinant Manufacturing System for Biopharmaceuticals .................................160
Chapter10 Upstream Processes Involved in Protein Production ................................................179
Chapter11 Downstream Processes Involved in Protein Production ...........................................228
Chapter12 Formulation of Biopharmaceuticals .........................................................................263
Chapter13 Quality and Compliance Systems ............................................................................307
Chapter14 Intellectual Property Issues for Scientists ................................................................343
Bibliography .................................................................................................................................377
Index ..............................................................................................................................................389
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vii
Preface
The rst chemically synthesized drug was chloral hydrate in 1832; aspirin was rst synthesized in
1899. Since then, millions of new drug molecules have been synthesized, many of which have ended
up as effective drugs, ranging from modulating immune systems to inactivating viruses, to treat
nearly every ailment. However, the discovery of insulin in 1922 revolutionized biological medicine.
Insulin was also the rst biopharmaceutical drug developed by applying recombinant technology.
Penicillin became available in 1928, thanks to Fleming and Waksman. The use of individual enzymatic transformation phases by employing microorganisms in chemical manufacturing pathways,
such as the biotransformation of steroids in 1950, expanded the scope of biotechnological pharmaceutical manufacturing.
The term “therapeutic protein” refers to the process of joining DNA obtained from two or more
different species to form the recombinant DNA (rDNA) product that is subsequently inserted
as the hybrid DNA into a host cell, often a bacterium or mammalian cell, to express the target
protein. Therapeutic proteins replace an abnormal or decient protein in a particular disease or
augment the supply of a benecial protein to the body to help reduce the effect of disease or chemotherapy. Genetically engineered proteins can closely resemble the natural proteins they replace
or be enhanced by adding sugars or other molecules in order to extend the protein’s duration of
activity. UC San Francisco and Stanford researchers created this molecular chimera in 1972. Stanley
Cohen of Stanford and Herbert Boyer of UCSF received a US patent in 1980. On July 26, 1974, ten
researchers, including six future Nobel Laureates (James Watson, Paul Berg, Stanley Cohen, David
Baltimore, Ronald Davis, and Daniel Nathans), wrote a letter to the magazine Science urging that
the National Institutes of Health should regulate rDNA technology.
The rst rDNA product came into light in 1982, which was when rDNA insulin was approved;
now, hundreds of recombinant proteins are approved by regulatory agencies. Examples of this diverse
class of compounds include interferons, cytokines, interleukins, thrombocytes, growth factors,
coagulation factors, blood factors, anticoagulants, Fc fusion proteins, monoclonal antibodies, etc.
The global biologics market is expected to reach an income of approximately USD 719.94 billion
by 2030, valued at USD 366.50 billion in 2021 and growing at a compound annual growth rate of
7.15% from 2022 to 2030. The current market of therapeutic proteins exceeds USD 380 billion.
The rst edition of the book, which is the rst book dedicated to biosimilars, was entitled,
Handbook of Biogeneric Therapeutic Proteins: Regulatory, Manufacturing, Testing, and Patent
Issues, published in early 2002, long before the term “biosimilar” came into vogue. Calling these
biologicals “biogeneric” was intentional to invoke a similar approval process as that currently available to generic chemical drugs. The United States (US) Food and Drug Administration (FDA)
recommended me that legal issues are involved when using the term “biogeneric” because “generic”
is associated with a specic Act of Congress. We selected the term “biosimilar” to indicate that these
products will be “biologically similar,” not necessarily chemically or otherwise. Because pharmacology, toxicology, and clinical responses to biological drugs are related to receptor binding, the
term “biosimilar” fulls the critical need for a clear denition. However, many regulatory agencies
use different labels, but currently, all of them seem to converge into one label, “biosimilars.”
Biosimilars evolved 18 years ago, and currently, they account for 76 products granted approvals
in the European Union (EU) and 41 in the US, and hundreds more worldwide. The European
Medicines Agency introduced the rst biosimilar guideline and approved the rst product in 2006;
the US guidelines came into use in 2019. Tables 1.2 and 1.3 list the approved (also rejected and
withdrawn) products in the US and EU.
Now that more than two decades have passed since I wrote the rst book on this subject, there
appears a need to revise the perspective presented earlier, as there may be changes in the views over
time, let alone for technology that is most rapidly advancing. Apart from technology, the regulatory
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Preface
process has also changed signicantly. The second edition, entitled, Handbook of Biosimilar
Therapeutic Proteins: Regulatory, Manufacturing, Testing, and Patent Issues, describes the major
changes, which would be of great importance to the developers, as did the rst edition of this book.
My initial goal was to introduce this technology to new developers, mostly in developing countries,
and this edition will be handy and continue to provide advice and suggestions on how to reduce
development cost, starting from the technology stage to the regulatory ling planning stage.
The rst edition was introduced by a dear friend, Francisco Baralle, who was the head of ICGEB,
Trieste, Italy, and was pivotal in developing and transferring recombinant technologies to many
countries. Francisco Baralle has left ICGEB, and I am pleased and honored to have him write an
introduction to this second edition.
I wish to express my gratitude to my scientic and professional colleagues, particularly to those
whom I know from the eld’s seminal literature and those whom I have never met. Given that I may
have unknowingly cited their research, assuming that it was available in the public domain, I hope
that I would be excused for taking this liberty, as it would be highly challenging for me to recognize
such citations. Finally, I also hope that I would be excused for any errors, as mentioned in the rst
edition of Encyclopedia Britannica (1786): “With regard to errors, in general, whether falling under
the denomination of mental, typographical, or accidental, I am conscious of being able to point out a
greater number than any critic. Men acquainted with the innumerable difculties attending the execution of a work of such an extensive nature will make proper allowances. To these, we appeal and
shall rest satised with the judgment they pronounce.”
I would appreciate your suggestions and comments, especially with regard to any errors in the
book, as they will help me to improve this treatise in the future.
Disclaimer: The author does not accept responsibility for any technical or legal suggestions or
advice provided in this book; all views expressed in this book are those of the author in his capacity
and not as the Patent Agent of the US Patent and Trademark Ofce, as an ofcer of any company or
in any academic positions held, or in any capacity as advisors to regulatory agencies.
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Author Biography
Sarfaraz K. Niazi, PhD, is an adjunct professor at the University
of Illinois. He has authored 60+ major books, 100+ research
papers, and 100+ patents, mainly in the eld of bioprocessing. He
has hands- on experience in biopharmaceutical projects, starting
from concept preparation to drug entry into the market, including
setting up the rst biosimilar company in the US and acquiring
several FDA approvals. He serves as an advisor to several regulatory agencies, including the FDA.
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