The Challenge of CMC Regulatory Compliance for Biopharmaceuticals

The Challenge of CMC Regulatory Compliance for Biopharmaceuticals

Author: John Geigert

Publisher: Springer Nature

Published: 2023-06-15

Total Pages: 597

ISBN-13: 3031319095

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Each year for the past three years, there have been about 50 new molecular medicines approved by the United States Food & Drug Administration (FDA), of which approximately 25% were new biopharmaceuticals. Over 200 recombinant proteins, monoclonal antibodies, antibody drug conjugates, fusion proteins, and Fab fragments are now in the marketplace in both the United States of America (USA) and European Union (EU). There are also now over 60 biosimilars available for all major classes of recombinant proteins and monoclonal antibodies. In addition, gene therapies using genetically engineered viruses and genetically engineered cells are now in the marketplace, and continually growing. This degree of change is reflected in the over 400 CMC regulatory compliance references listed in this book that were either issued or updated since the release of the third edition. Deficiencies in biopharmaceutical CMC regulatory compliance rarely result in termination of a product, but in can readily cause months if not years of delay in initiating clinical trials, or advancing clinical development stages, or even market approval. In summary, this book: Updates real-world CMC deficiency examples with current examples; Addresses current FDA and EMA requirements and expectations for CMC regulatory compliance; Now includes CMC regulatory compliance for the new gene-based biopharmaceuticals.


Book Synopsis The Challenge of CMC Regulatory Compliance for Biopharmaceuticals by : John Geigert

Download or read book The Challenge of CMC Regulatory Compliance for Biopharmaceuticals written by John Geigert and published by Springer Nature. This book was released on 2023-06-15 with total page 597 pages. Available in PDF, EPUB and Kindle. Book excerpt: Each year for the past three years, there have been about 50 new molecular medicines approved by the United States Food & Drug Administration (FDA), of which approximately 25% were new biopharmaceuticals. Over 200 recombinant proteins, monoclonal antibodies, antibody drug conjugates, fusion proteins, and Fab fragments are now in the marketplace in both the United States of America (USA) and European Union (EU). There are also now over 60 biosimilars available for all major classes of recombinant proteins and monoclonal antibodies. In addition, gene therapies using genetically engineered viruses and genetically engineered cells are now in the marketplace, and continually growing. This degree of change is reflected in the over 400 CMC regulatory compliance references listed in this book that were either issued or updated since the release of the third edition. Deficiencies in biopharmaceutical CMC regulatory compliance rarely result in termination of a product, but in can readily cause months if not years of delay in initiating clinical trials, or advancing clinical development stages, or even market approval. In summary, this book: Updates real-world CMC deficiency examples with current examples; Addresses current FDA and EMA requirements and expectations for CMC regulatory compliance; Now includes CMC regulatory compliance for the new gene-based biopharmaceuticals.

The Challenge of CMC Regulatory Compliance for Biopharmaceuticals

The Challenge of CMC Regulatory Compliance for Biopharmaceuticals

Author: John Geigert

Publisher: Springer Science & Business Media

Published: 2014-07-08

Total Pages: 362

ISBN-13: 1461469163

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This book highlights the challenges facing quality assurance/quality control (QA/QC) in today's biopharmaceutical environment and presents the strategic importance and value generated by QA/QC for their involvement in control of manufacturing. It will put into perspective the need for a graded approach to QA/QC from early clinical trials through market approval. Since the first edition published in 2004, there have been more than 50 new regulatory guidances released by the Food and Drug Administration (FDA), European Medicines Agency (EMA) and ICH that affect the CMC regulatory compliance of biopharmaceuticals; also the application of biosimilars has been developed in Europe and is under development in the USA. The revised update will be broadened to include not only biopharmaceuticals (biotech drugs) but also other biologics (vaccines, cell therapy, plasma-derived proteins, etc.)


Book Synopsis The Challenge of CMC Regulatory Compliance for Biopharmaceuticals by : John Geigert

Download or read book The Challenge of CMC Regulatory Compliance for Biopharmaceuticals written by John Geigert and published by Springer Science & Business Media. This book was released on 2014-07-08 with total page 362 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book highlights the challenges facing quality assurance/quality control (QA/QC) in today's biopharmaceutical environment and presents the strategic importance and value generated by QA/QC for their involvement in control of manufacturing. It will put into perspective the need for a graded approach to QA/QC from early clinical trials through market approval. Since the first edition published in 2004, there have been more than 50 new regulatory guidances released by the Food and Drug Administration (FDA), European Medicines Agency (EMA) and ICH that affect the CMC regulatory compliance of biopharmaceuticals; also the application of biosimilars has been developed in Europe and is under development in the USA. The revised update will be broadened to include not only biopharmaceuticals (biotech drugs) but also other biologics (vaccines, cell therapy, plasma-derived proteins, etc.)

Peptide Therapeutics

Peptide Therapeutics

Author: Ved Srivastava

Publisher: Royal Society of Chemistry

Published: 2019-08-28

Total Pages: 572

ISBN-13: 1788014332

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Peptide therapy has become a key strategy in innovative drug development, however, one of the potential barriers for the development of novel peptide drugs in the clinic is their deficiencies in clearly defined chemistry, manufacturing and controls (CMC) strategy from clinical development to commercialization. CMC can often become a rate-limiting step due to lack of knowledge and lack of a formal policy or guidelines on CMC for peptide-based drugs. Regulators use a risk-based approach, reviewing applications on a case-by-case basis. Peptide Therapeutics: Strategy and Tactics for Chemistry, Manufacturing, and Controls covers efficient manufacturing of peptide drug substances, a review of the process for submitting applications to the regulatory authority for drug approval, a holistic approach for quality attributes and quality control from a regulatory perspective, emerging analytical tools for the characterisation of impurities, and the assessment of stability. This book is an essential reference work for students and researchers, in both academia and industry, with an interest in learning about CMC, and facilitating development and manufacture of peptide-based drugs.


Book Synopsis Peptide Therapeutics by : Ved Srivastava

Download or read book Peptide Therapeutics written by Ved Srivastava and published by Royal Society of Chemistry. This book was released on 2019-08-28 with total page 572 pages. Available in PDF, EPUB and Kindle. Book excerpt: Peptide therapy has become a key strategy in innovative drug development, however, one of the potential barriers for the development of novel peptide drugs in the clinic is their deficiencies in clearly defined chemistry, manufacturing and controls (CMC) strategy from clinical development to commercialization. CMC can often become a rate-limiting step due to lack of knowledge and lack of a formal policy or guidelines on CMC for peptide-based drugs. Regulators use a risk-based approach, reviewing applications on a case-by-case basis. Peptide Therapeutics: Strategy and Tactics for Chemistry, Manufacturing, and Controls covers efficient manufacturing of peptide drug substances, a review of the process for submitting applications to the regulatory authority for drug approval, a holistic approach for quality attributes and quality control from a regulatory perspective, emerging analytical tools for the characterisation of impurities, and the assessment of stability. This book is an essential reference work for students and researchers, in both academia and industry, with an interest in learning about CMC, and facilitating development and manufacture of peptide-based drugs.

Regulatory Aspects of Gene Therapy and Cell Therapy Products

Regulatory Aspects of Gene Therapy and Cell Therapy Products

Author: Maria Cristina Galli

Publisher: Springer Nature

Published: 2023-08-01

Total Pages: 243

ISBN-13: 3031345673

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This book discusses the different regulatory pathways for Advanced Therapy Medicinal Products implemented by national agencies in North and South America, Europe and Asia and by international bodies in the effort of international harmonization. This book represents an update of the first edition, as it covers regulatory novelties and accumulated experience in the regions already addressed. In addition, this new edition offers a wider international perspective: new chapters are included covering Advanced Therapy Medicinal Products regulations in India, Malaysia, Spain and Thailand, the European Pharmacopoeia texts for gene therapy medicinal products as well as international harmonization programs. Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure and therapeutic use of these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of Advanced Therapy Medicinal Products in the areas of product quality, pharmacology and toxicology, clinical trial design and HTA pathways, as well as pertinent ‘must-know’ guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: a Global Perspective is part of the American Society of Gene & Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.


Book Synopsis Regulatory Aspects of Gene Therapy and Cell Therapy Products by : Maria Cristina Galli

Download or read book Regulatory Aspects of Gene Therapy and Cell Therapy Products written by Maria Cristina Galli and published by Springer Nature. This book was released on 2023-08-01 with total page 243 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book discusses the different regulatory pathways for Advanced Therapy Medicinal Products implemented by national agencies in North and South America, Europe and Asia and by international bodies in the effort of international harmonization. This book represents an update of the first edition, as it covers regulatory novelties and accumulated experience in the regions already addressed. In addition, this new edition offers a wider international perspective: new chapters are included covering Advanced Therapy Medicinal Products regulations in India, Malaysia, Spain and Thailand, the European Pharmacopoeia texts for gene therapy medicinal products as well as international harmonization programs. Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure and therapeutic use of these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of Advanced Therapy Medicinal Products in the areas of product quality, pharmacology and toxicology, clinical trial design and HTA pathways, as well as pertinent ‘must-know’ guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: a Global Perspective is part of the American Society of Gene & Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

Bioprocessing for Biomolecules Production

Bioprocessing for Biomolecules Production

Author: Gustavo Molina

Publisher: John Wiley & Sons

Published: 2020-01-21

Total Pages: 532

ISBN-13: 1119434327

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Presents the many recent innovations and advancements in the field of biotechnological processes This book tackles the challenges and potential of biotechnological processes for the production of new industrial ingredients, bioactive compounds, biopolymers, energy sources, and compounds with commercial/industrial and economic interest by performing an interface between the developments achieved in the recent worldwide research and its many challenges to the upscale process until the adoption of commercial as well as industrial scale. Bioprocessing for Biomolecules Production examines the current status of the use and limitation of biotechnology in different industrial sectors, prospects for development combined with advances in technology and investment, and intellectual and technical production around worldwide research. It also covers new regulatory bodies, laws and regulations, and more. Chapters look at biological and biotechnological processes in the food, pharmaceutical, and biofuel industries; research and production of microbial PUFAs; organic acids and their potential for industry; second and third generation biofuels; the fermentative production of beta-glucan; and extremophiles for hydrolytic enzymes productions. The book also looks at bioethanol production from fruit and vegetable wastes; bioprocessing of cassava stem to bioethanol using soaking in aqueous ammonia pretreatment; bioprospecting of microbes for bio-hydrogen production; and more. Provides up to date information about the advancements made on the production of important biotechnological ingredients Complete visualization of the general developments of world research around diverse products and ingredients of technological, economic, commercial and social importance Investigates the use and recovery of agro-industrial wastes in biotechnological processes Includes the latest updates from regulatory bodies for commercialization feasibility Offering new products and techniques for the industrial development and diversification of commercial products, Bioprocessing for Biomolecules Production is an important book for graduate students, professionals, and researchers involved in food technology, biotechnology; microbiology, bioengineering, biochemistry, and enzymology.


Book Synopsis Bioprocessing for Biomolecules Production by : Gustavo Molina

Download or read book Bioprocessing for Biomolecules Production written by Gustavo Molina and published by John Wiley & Sons. This book was released on 2020-01-21 with total page 532 pages. Available in PDF, EPUB and Kindle. Book excerpt: Presents the many recent innovations and advancements in the field of biotechnological processes This book tackles the challenges and potential of biotechnological processes for the production of new industrial ingredients, bioactive compounds, biopolymers, energy sources, and compounds with commercial/industrial and economic interest by performing an interface between the developments achieved in the recent worldwide research and its many challenges to the upscale process until the adoption of commercial as well as industrial scale. Bioprocessing for Biomolecules Production examines the current status of the use and limitation of biotechnology in different industrial sectors, prospects for development combined with advances in technology and investment, and intellectual and technical production around worldwide research. It also covers new regulatory bodies, laws and regulations, and more. Chapters look at biological and biotechnological processes in the food, pharmaceutical, and biofuel industries; research and production of microbial PUFAs; organic acids and their potential for industry; second and third generation biofuels; the fermentative production of beta-glucan; and extremophiles for hydrolytic enzymes productions. The book also looks at bioethanol production from fruit and vegetable wastes; bioprocessing of cassava stem to bioethanol using soaking in aqueous ammonia pretreatment; bioprospecting of microbes for bio-hydrogen production; and more. Provides up to date information about the advancements made on the production of important biotechnological ingredients Complete visualization of the general developments of world research around diverse products and ingredients of technological, economic, commercial and social importance Investigates the use and recovery of agro-industrial wastes in biotechnological processes Includes the latest updates from regulatory bodies for commercialization feasibility Offering new products and techniques for the industrial development and diversification of commercial products, Bioprocessing for Biomolecules Production is an important book for graduate students, professionals, and researchers involved in food technology, biotechnology; microbiology, bioengineering, biochemistry, and enzymology.

Vaccines

Vaccines

Author: Stanley A. Plotkin

Publisher: Elsevier Health Sciences

Published: 2008-02-11

Total Pages: 1748

ISBN-13: 1437721583

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Completely revised and updated, this respected reference offers comprehensive and current coverage of every aspect of vaccination-from development to use in reducing disease. It provides authoritative information on vaccine production, available preparations, efficacy, and safety...recommendations for vaccine use, with rationales...data on the impact of vaccination programs on morbidity and mortality...and more. And now, as an Expert Consult title, it includes a companion web site offering this unparalleled guidance where and when you need it most! Provides a complete understanding of each disease, including clinical characteristics, microbiology, pathogenesis, diagnosis, and treatment, as well an epidemiology and public health issues. Offers comprehensive coverage of both existing vaccines and vaccines currently in the research and development stage. Examines vaccine stability, immunogenicity, efficacy, duration of immunity, adverse events, indications, contraindications, precautions, administration with other vaccines, and disease control strategies. Analyses the cost-benefit and cost-effectiveness of vaccines. Discusses the proper use of immune globulins and antitoxins. Illustrates concepts and objective data with approximately 600 tables and figures. Includes access to a companion web site offering the complete contents of the book - fully searchable - for rapid consultation from anyplace with an Internet connection.


Book Synopsis Vaccines by : Stanley A. Plotkin

Download or read book Vaccines written by Stanley A. Plotkin and published by Elsevier Health Sciences. This book was released on 2008-02-11 with total page 1748 pages. Available in PDF, EPUB and Kindle. Book excerpt: Completely revised and updated, this respected reference offers comprehensive and current coverage of every aspect of vaccination-from development to use in reducing disease. It provides authoritative information on vaccine production, available preparations, efficacy, and safety...recommendations for vaccine use, with rationales...data on the impact of vaccination programs on morbidity and mortality...and more. And now, as an Expert Consult title, it includes a companion web site offering this unparalleled guidance where and when you need it most! Provides a complete understanding of each disease, including clinical characteristics, microbiology, pathogenesis, diagnosis, and treatment, as well an epidemiology and public health issues. Offers comprehensive coverage of both existing vaccines and vaccines currently in the research and development stage. Examines vaccine stability, immunogenicity, efficacy, duration of immunity, adverse events, indications, contraindications, precautions, administration with other vaccines, and disease control strategies. Analyses the cost-benefit and cost-effectiveness of vaccines. Discusses the proper use of immune globulins and antitoxins. Illustrates concepts and objective data with approximately 600 tables and figures. Includes access to a companion web site offering the complete contents of the book - fully searchable - for rapid consultation from anyplace with an Internet connection.

Cell and Gene Therapies

Cell and Gene Therapies

Author: Miguel-Angel Perales

Publisher: Springer

Published: 2018-11-27

Total Pages: 288

ISBN-13: 3319543687

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In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, including hematopoietic transplant physicians, with information that is relevant to clinical practice and ongoing research. Each chapter focuses on a particular topic, and the concise text is supported by numerous working tables, algorithms, and figures. Whenever appropriate, guidance is provided regarding the availability of potentially high-impact clinical trials. The rapid evolution of cell and gene therapies is giving rise to numerous controversies that need to be carefully addressed. In meeting this challenge, this book will appeal to all residents, fellows, and faculty members responsible for the care of hematopoietic cell transplant patients. It will also offer a robust, engaging tool to aid vital activities in the daily work of every hematology and oncology trainee.


Book Synopsis Cell and Gene Therapies by : Miguel-Angel Perales

Download or read book Cell and Gene Therapies written by Miguel-Angel Perales and published by Springer. This book was released on 2018-11-27 with total page 288 pages. Available in PDF, EPUB and Kindle. Book excerpt: In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, including hematopoietic transplant physicians, with information that is relevant to clinical practice and ongoing research. Each chapter focuses on a particular topic, and the concise text is supported by numerous working tables, algorithms, and figures. Whenever appropriate, guidance is provided regarding the availability of potentially high-impact clinical trials. The rapid evolution of cell and gene therapies is giving rise to numerous controversies that need to be carefully addressed. In meeting this challenge, this book will appeal to all residents, fellows, and faculty members responsible for the care of hematopoietic cell transplant patients. It will also offer a robust, engaging tool to aid vital activities in the daily work of every hematology and oncology trainee.

Handbook of Stability Testing in Pharmaceutical Development

Handbook of Stability Testing in Pharmaceutical Development

Author: Kim Huynh-Ba

Publisher: Springer Science & Business Media

Published: 2008-11-16

Total Pages: 389

ISBN-13: 0387856277

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This handbook is the first to cover all aspects of stability testing in pharmaceutical development. Written by a group of international experts, the book presents a scientific understanding of regulations and balances methodologies and best practices.


Book Synopsis Handbook of Stability Testing in Pharmaceutical Development by : Kim Huynh-Ba

Download or read book Handbook of Stability Testing in Pharmaceutical Development written by Kim Huynh-Ba and published by Springer Science & Business Media. This book was released on 2008-11-16 with total page 389 pages. Available in PDF, EPUB and Kindle. Book excerpt: This handbook is the first to cover all aspects of stability testing in pharmaceutical development. Written by a group of international experts, the book presents a scientific understanding of regulations and balances methodologies and best practices.

Development and Manufacture of Protein Pharmaceuticals

Development and Manufacture of Protein Pharmaceuticals

Author: Steve L. Nail

Publisher: Springer Science & Business Media

Published: 2012-12-06

Total Pages: 479

ISBN-13: 1461505496

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In this era of biotechnology there have been many books covering the fundamentals of recombinant DNA technology and protein chemistry. However, not many sources are available for the pharmaceutical develop ment scientist and other personnel responsible for the commercialization of the finished dosage forms of these new biopharmaceuticals and other products from biotechnology. This text will help to fill this gap. Once active biopharmaceutical molecules are candidates for clinical trial investigation and subsequent commercialization, a number of other activities must take place while research and development on these molecules continues. The active ingredient itself must be formulated into a finished dosage form that can be conveniently used by health care professionals and patients. Properties of the biopharmaceutical molecule must be clearly understood so that the appropriate finished product formulation can be developed. Finished product formulation development includes not only the chemical formulation, but also the packaging system, the manufacturing process, and appropriate control strategies to assure such good manufacturing practice attributes as safety, identity, strength, purity, and quality.


Book Synopsis Development and Manufacture of Protein Pharmaceuticals by : Steve L. Nail

Download or read book Development and Manufacture of Protein Pharmaceuticals written by Steve L. Nail and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 479 pages. Available in PDF, EPUB and Kindle. Book excerpt: In this era of biotechnology there have been many books covering the fundamentals of recombinant DNA technology and protein chemistry. However, not many sources are available for the pharmaceutical develop ment scientist and other personnel responsible for the commercialization of the finished dosage forms of these new biopharmaceuticals and other products from biotechnology. This text will help to fill this gap. Once active biopharmaceutical molecules are candidates for clinical trial investigation and subsequent commercialization, a number of other activities must take place while research and development on these molecules continues. The active ingredient itself must be formulated into a finished dosage form that can be conveniently used by health care professionals and patients. Properties of the biopharmaceutical molecule must be clearly understood so that the appropriate finished product formulation can be developed. Finished product formulation development includes not only the chemical formulation, but also the packaging system, the manufacturing process, and appropriate control strategies to assure such good manufacturing practice attributes as safety, identity, strength, purity, and quality.

Biosimilars

Biosimilars

Author: Hiten J. Gutka

Publisher: Springer

Published: 2018-12-13

Total Pages: 709

ISBN-13: 3319996800

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This book provides a comprehensive overview of the biosimilar regulatory framework, the development process and clinical aspects for development of biosimilars. The development path of a biosimilar is just as unique as a development path of a new drug, tailored by the mechanism of action, the quality of the molecule, published information on the reference product, the current competitive environment, the target market and regulatory guidance, and most importantly, the emerging totality of evidence for the proposed biosimilar during development. For the ease of readers, the book comprises of six sections as follows: Section I: Business, Health Economics and Intellectual Property Landscape for Biosimilars Section II: Regulatory Aspects of Development and Approval for Biosimilars Section III: Biopharmaceutical Development and Manufacturing of Biosimilars Section IV: Analytical Similarity Considerations for Biosimilars Section V: Clinical aspects of Biosimilar Development Section VI: Biosimilars- Global Development and Clinical Experience Chapters have been written by one or more experts from academia, industry or regulatory agencies who have been involved with one or more aspects of biosimilar product development. The authors and editors have an expertise in commercialization and pricing of biosimilars, intellectual property considerations for biosimilars, chemistry manufacturing controls (CMC) and analytical development for biosimilars, regulatory and clinical aspects of biosimilar development. Besides the industry practitioners, the book includes several contributions from regulators across the globe.


Book Synopsis Biosimilars by : Hiten J. Gutka

Download or read book Biosimilars written by Hiten J. Gutka and published by Springer. This book was released on 2018-12-13 with total page 709 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book provides a comprehensive overview of the biosimilar regulatory framework, the development process and clinical aspects for development of biosimilars. The development path of a biosimilar is just as unique as a development path of a new drug, tailored by the mechanism of action, the quality of the molecule, published information on the reference product, the current competitive environment, the target market and regulatory guidance, and most importantly, the emerging totality of evidence for the proposed biosimilar during development. For the ease of readers, the book comprises of six sections as follows: Section I: Business, Health Economics and Intellectual Property Landscape for Biosimilars Section II: Regulatory Aspects of Development and Approval for Biosimilars Section III: Biopharmaceutical Development and Manufacturing of Biosimilars Section IV: Analytical Similarity Considerations for Biosimilars Section V: Clinical aspects of Biosimilar Development Section VI: Biosimilars- Global Development and Clinical Experience Chapters have been written by one or more experts from academia, industry or regulatory agencies who have been involved with one or more aspects of biosimilar product development. The authors and editors have an expertise in commercialization and pricing of biosimilars, intellectual property considerations for biosimilars, chemistry manufacturing controls (CMC) and analytical development for biosimilars, regulatory and clinical aspects of biosimilar development. Besides the industry practitioners, the book includes several contributions from regulators across the globe.