Analytical similarity as the primary determinant of monoclonal antibody development: toward evidence-efficient regulatory frameworks

分析相似性作为单克隆抗体研发的主要决定因素:迈向循证高效的监管框架

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Abstract

Monoclonal antibodies (mAbs) constitute the most rapidly expanding and therapeutically impactful class of biological medicines, driven by their exceptional target specificity, modular engineering capabilities, predictable pharmacokinetics facilitated by neonatal Fc receptor (FcRn) recycling, and favorable safety profiles attributable to mechanism-based pharmacology. As foundational patents on blockbuster antibody therapeutics expire, biosimilar antibodies have become a vital mechanism to enhance global accessibility, improve healthcare sustainability, and promote market competition. Simultaneously, regulatory agencies worldwide are substantially restructuring development expectations to emphasize analytical precision, mechanistic understanding, human-relevant methodologies, and ethical nonclinical practices aligned with the 3Rs principles of replacement, reduction, and refinement. This comprehensive review analyzes the convergence of innovator and biosimilar antibody development toward an analytics-first comparability paradigm, mechanism-based safety assessment, and targeted clinical confirmation based on residual uncertainty rather than prescriptive requirements. It systematically incorporates regulatory positions from the United States Food and Drug Administration (FDA), European Medicines Agency (EMA), World Health Organization (WHO), Japan's Pharmaceuticals and Medical Devices Agency (PMDA), Health Canada, and the International Council for Harmonisation (ICH), together with contemporary peer-reviewed research literature. Focus is given to the FDA Modernization Act 2.0, which abolished statutory mandates for animal testing, and recent FDA draft guidance-which are provisional and open to revision pending finalization-addressing streamlined nonclinical safety studies for monospecific antibodies and science-based approaches to waiving comparative clinical efficacy studies (CES). The scientific boundaries distinguishing products amenable to streamlined development from those requiring tailored approaches are delineated, including the exclusion of polyclonal antibody preparations from biosimilar frameworks and the analytical complexities associated with conjugated antibodies and multispecific constructs. Important limitations of analytics-first approaches are also addressed, including scenarios involving heightened immunogenicity risk, novel targets with limited clinical experience, or insufficiently validated pharmacodynamic markers. These developments are examined alongside counterarguments and residual areas of regulatory skepticism. While the trajectory of regulatory reform appears to favor evidence-efficient, science-driven models for antibody development, this review explicitly distinguishes between established regulatory consensus, emerging draft guidance positions, and the author's scholarly interpretation, recognizing that the pace and scope of regulatory change remain subject to ongoing deliberation.

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