Module 9 2024

03/09/2024

Recommendations, AAPS White Papers & Others (ctd)

Report on the AAPS Immunogenicity Guidance Forum (2019). Myler H. et al., AAPS J, 21(4):55-61

Current Considerations for Immunoglobulin Isotype Characterization of Antibody Response against Biotherapeutics (2020). Gorovits B., AAPS J. 22(6):144

A strategic approach to nonclinical immunogenicity assessment: a recommendation from the European Bioanalysis Forum (2021). Laurén A. et al., Bioanalysis, 13(7): 537-549

When to Extend Monitoring of Anti-drug Antibodies for High-risk Biotherapeutics in Clinical Trials: an Opinion from the European Immunogenicity Platform (2022). Lotz G. et al., AAPS J. 24(68): https://doi.org/10.1208/s12248-022-00712-2

Anti-drug Antibody validation Testing and Reporting harmonization (2022). Myler H. et al, AAPS J. 24(4): https://doi.org/10.1208/s12248-021-00649-y

Immunogenicity assessment of AAV-based gene therapies: An IQ Consortium industry White Paper (2022). Yang T.Y. et al, Molecular Therapy: Methods & Clinical Development 26:471-494

2023 White Paper on Recent Issues in Bioanalysis: ISR for ADA Assays, the Rise of dPCR vs qPCR, International Reference Standards for Vaccine Assays, Anti-AAV TAb Post-Dose Assessment, NanoString Validation, ELISpot as Gold Standard (Part 3 - Recommendations on Gene Therapy, Cell Therapy, Vaccines Immunogenicity & Technologies; Biotherapeutics Immunogenicity & Risk Assessment; ADA/NAb Assay/Reporting Harmonization) (2024). Mora J. et al, Bioanalysis 6(7): 77 – 119

Neutralizing Antibody Sample Testing and Report Harmonization (2024), Jani D. et al, AAPS J. 26(80) https://doi.org/10.1208/s12248-024-00955-1

The Organisation for Professionals in Regulatory Affairs

39

Learning Outcomes

What is immunogenicity? - Definitions

Why / when does it occur? - Factors involved

Strategy for immunogenicity assessment - Tiered approach

Screening & confirmatory immunoassays - ELISA, ECLA

Neutralising antibody assays - Bioassays, CLBA

Issues & considerations - Overview of potential assay problems

Recommendations & Guidelines - EMA, FDA, White Papers

The Organisation for Professionals in Regulatory Affairs

40

20