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Immunogenicity Testing of Therapeutic Protein Products —Developing and Validating Assays for Anti-Drug Antibody Detection

FinalCenter for Drug Evaluation and Research Center for Biologics Evaluation and Research02/01/2019

Description

The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Immunogenicity Testing of Therapeutic Protein Products—Developing and Validating Assays for Anti-Drug Antibody Detection.” This guidance provides recommendations to facilitate industry's development and validation of assays for assessment of the immunogenicity of therapeutic protein products during clinical trials. The guidance applies to assays for the detection of anti-drug antibodies (ADAs) and may also apply to some peptides, oligonucleotides, and combination products on a case-by-case basis. The guidance includes recommendations regarding the development and validation of screening assays, confirmatory assays, titration assays, and neutralization assays. This guidance finalizes the revised draft guidance for industry entitled “Assay Development and Validation for Immunogenicity Testing of Therapeutic Protein Products” issued in April 2016 and includes a revised title.

Scope & Applicability

Product Classes

8
Therapeutic Protein Product

Focus of the guidance for determining when a CES may inform biosimilarity

mAb products

especially important for mAb products because these products can have half-lives of several weeks

Antibody-Drug Conjugates

Specific type of therapeutic protein discussed for DDIs; Focus of biologics DDI evaluation.; ADCs consist of a small molecule drug component conjugated to an antibody.

Biosimilar

Biological product shown to be highly similar to an FDA-licensed reference product; Biological product demonstrated to be highly similar to a reference product

Therapeutic Protein Products

Guidance on immunogenicity assessment and testing

Combination Product

Products combining drug, device, or biological constituents; Generally recommended for Enhanced Documentation; Requires 14971-based framework incorporating ICH Q9; A drug-device combination where the device constituent part detects ingestion.

In Vitro Diagnostic Products

Licensed IVDs subject to stability requirements

Bispecific Antibodies

Multi-domain products requiring multiple assays for different domains.

Stakeholders

2
treatment-naïve subjects

Samples from these individuals are used to statistically determine cut-points; Samples from treatment-naïve subjects that are collected, handled, and stored under study conditions.; samples from treatment-naïve subjects used for cut-point

Sponsor

Entity responsible for submitting applications under section 524B

Regulatory Context

Attributes

9
Sensitivity

Analysis by sex of clinical performance measures such as sensitivity

Titers

Measurement of antibody level based on risk

Half-life

Property used to determine maintenance dose intervals and safety monitoring

Percent coefficient of variation

Intra-assay and inter-assay precision as expressed by percent coefficient of variation (%CV) is expected to be lower than 20%.

False-positive rate

A rate of approximately 5% is desirable for the initial screening assay.; One approach that allows for high assurance of a 5% false-positive rate.

Minimal Required Dilution

Factor used to minimize matrix interference, recommended not to exceed 1:100

Specificity

Ability to detect intended mechanism of action without interference; Performance characteristic to be validated

Selectivity

Ability to differentiate analyte in presence of matrix components

Drug Tolerance

A key operating parameter of the assay affecting ADA incidence.; Performance characteristic to be established between laboratories

Identified Hazards

Hazards

3
Matrix Interference

Signal suppression or non-specific signal caused by endogenous components

Prozone effects

If the assay is subject to a prozone effect, the concentration of high-positive QC samples should be set

Hook Effects

Reduction in signal caused by high concentration of analyte leading to false-negatives

Related CFR Sections (2)

See Also (8)