Biopharmaceuticals (i.e., biological medicines sourced from
genetically-engineered living systems) for treatment of human diseases
have become a significant percentage of the pharmaceutical industry. And
not just the recombinant DNA-derived proteins and monoclonal antibodies
(both from the innovators and biosimilars); but now, an increasing
awareness of the importance of gene therapy and genetically engineered
cellular medicinal products. These biopharmaceuticals are being
developed by many companies whose Chemistry, Manufacturing & Control
(CMC) teams have varying degrees of familiarity or experience with the
CMC strategy and regulatory compliance requirements for these
challenging products. Companies clearly plan out the strategy for their
clinical study plans, but frequently, the development of a strategy for
CMC is an afterthought. Coupled with the complexity of the
biopharmaceutical manufacturing processes and products, and this can be
a recipe for disaster. The third edition of this book provides insights
and practical guidance for the CMC teams to develop an acceptable
cost-effective, risk-based CMC regulatory compliance strategy for all
biopharmaceuticals (recombinant proteins, monoclonal antibodies,
genetically engineered viruses and genetically engineered human cells)
from early clinical stage development through market approval. The third
edition of this book provides added coverage for the biosimilars,
antibody drug conjugates (ADCs), bispecific antibodies, genetically
engineered viruses, and genetically engineered cells. This third edition
of the book also addresses the heightened pressure on CMC regulatory
compliance timelines due to the introduction of expedited clinical
pathways moving the clinical development closer to a seamless phase
process (e.g., FDA Breakthrough Therapy designation, CBER Regenerative
Medicine Advanced Therapy (RMAT) designation, EMA Priority Medicines
(PRIME) designation). The Challenge of CMC Regulatory Compliance for
Biopharmaceuticals is essential, practical information for all
pharmaceutical development scientists, Manufacturing and Quality Unit
staff, Regulatory Affairs personnel, and senior management involved in
the manufacture of biopharmaceuticals.
