Back to Explorer

Questions and Answers | M7(R2) Assessment and Control of DNA Reactive (Mutagenic) Impurities in Pharmaceuticals To Limit Potential Carcinogenic Risk: Guidance for Industry

FinalCenter for Drug Evaluation and Research Center for Biologics Evaluation and Research07/25/2023
Hazard AssessmentOption 4 Control StrategiesRisk CharacterizationControl Strategy

Description

The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “M7(R2) Assessment and Control of DNA Reactive (Mutagenic) Impurities in Pharmaceuticals to Limit Potential Carcinogenic Risk” (M7(R2) Guidance) and two supplemental documents entitled “M7(R2) Addendum: Application of the Principles of the ICH M7 Guidance to Calculation of Compound-Specific Acceptable Intakes” (M7(R2) Addendum) and “M7(R2) Assessment and Control of DNA Reactive (Mutagenic) Impurities in Pharmaceuticals to Limit Potential Carcinogenic Risk Questions and Answers” (M7(R2) Questions and Answers). The M7(R2) Guidance, M7(R2) Addendum, and M7(R2) Questions and Answers were prepared under the auspices of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH). The guidance and supplemental documents are intended to harmonize the considerations for assessment and control of DNA reactive (mutagenic) impurities.

Key Topics

Terms and concepts identified from this document

Scope & Applicability

Product Classes

3
Pharmaceuticals

Scope of the mutagenic impurities assessment; AI calculated for potential pharmaceutical impurities; General context of ICH guidelines for industry; benzyl chloride as an impurity in drugs; Hydrazine is used in the synthesis of these products; Derivation of an AI for inhaled pharmaceuticals; Application of limits in pharmaceutical products; Application of ICH M7 principles to pharmaceutical impurities.

Anticancer Pharmaceuticals

Pharmaceuticals intended to treat cancer in patients with serious and life-threatening malignancies

Semisynthetic drug substances

Guidance applies to synthetic steps in semisynthetic drug substances

Stakeholders

2
Sponsor

Entity responsible for submitting applications under section 524B

DMF holder

new facilities added by the DMF holder

Regulatory Context

Regulatory Activities

2
Marketing authorization applications

Regulatory submission requiring assessment of impurities

Marketing Application

Submission for product approval

Document Types

5
Master files

Applicable to regulatory submissions.

(Q)SAR Model Reporting Format

OECD (Q)SAR Model Reporting Format (QMRF)

OECD (Q)SAR Model Reporting Format

QMRF template for reporting model validation

Drug Master File

A submission to FDA that may be used to provide confidential information about facilities or processes.; cross-referencing a DMF in a BLA

Common Technical Document

Format for submitting development information; Module 3 of the CTD for detailed descriptions of analytical procedures; ICH guideline M4Q(R1) for the registration of pharmaceuticals.

Attributes

2
1 mg daily dose

Threshold for expectations of mutagenic potential evaluation

Treatment duration

Scenario of >10 years to lifetime

Technical Details

Substances

4
Degradation products

Formed in the drug product

DNA Reactive (Mutagenic) Impurities

Assessment and control of these substances to limit carcinogenic risk; Impurities that reside in drug substances and drug products; Impurities that induce direct DNA damage through chemical reaction; Assessment and control of mutagenic impurities

Active pharmaceutical ingredient

Substance intended for incorporation into a finished drug product

Mutagenic Impurities

DNA reactive impurities in pharmaceuticals to limit carcinogenic risk; DNA reactive impurities to be controlled below acceptable limits

Testing Methods

7
Bacterial reverse mutation assay

Ames test used as main criterion for determining mutagenicity; Ames test used to evaluate DMS mutagenicity; Ames test used for mutagenicity testing

(Q)SAR Analyses

Quantitative Structure-Activity Relationship analyses used for mutagenic impurity assessment

(Q)SAR

quantitative structure-activity relationship assessment

Chromosomal aberration test

Used to evaluate genotoxic potential/clastogenicity

Predictive Purge Calculations

Scientific principles used to justify Option 4 control

Bacterial Reverse Mutagenicity Assay

Used to update (Q)SAR models and assess hazard

Periodic Verification Testing

Also known as skip testing per ICH Q6A; Also known as skip testing in ICH Q6A

Processes

3
Drug development

Phase during which information on impurities should be provided

Drug Substance Synthesis

Changes resulting in new impurities

Recrystallization

Purification process involving solvents

Clinical Concepts

1
HIV disease

treatment duration category changed due to clinical advances

Identified Hazards

Hazards

4
Carcinogenic Risk

Potential risk limited by controlling mutagenic impurities; Potential risk from DNA reactive impurities; Limit potential carcinogenic risk from impurities

Mutagenic Impurities

DNA Reactive Impurities in Pharmaceuticals

Mutagenic potential

Ability of a compound to induce point mutations

Genotoxic potential

Refers to mutagenic, clastogenic, or aneugenic potential

Standards & References

External Standards

1
OECD Validation 2007

General validation principles for (Q)SAR models

Specifications

3
Threshold of toxicological concern

TTC used to control impurities; TTC used for impurity risk assessment

Class 5

Classification for impurities with negative mutagenicity results

Acceptable Intake

AI limits for mutagenic impurities

ICH References (10)

ICH M7(R2)

Assessment and Control of DNA Reactive (Mutagenic) Impurities in Pharmaceuticals to Limit Potential Carcinogenic Risk; Application of the principles of the ICH M7 guideline to calculation of compound-specific acceptable intakes; ICH guidance for industry M7(R2) Assessment and Control of DNA Reactive (Mutagenic) Impurities; Document title and version; Document title and addendum reference; Guidance for assessment and control of DNA reactive impurities.; Assessment and Control of DNA Reactive Impu

ICH S9

Nonclinical Evaluation for Anticancer Pharmaceuticals; Guideline regarding pharmaceuticals for advanced cancer.

ICH M7(R1)

Assessment and Control of DNA Reactive (Mutagenic) Impurities in Pharmaceuticals to Limit Potential Carcinogenic Risk

ICH M4S

The CTD — Safety

ICH M4Q

The CTD — Quality

ICH M4

Organization of the Common Technical Document; Organization of the Common Technical Document for the Registration of Pharmaceuticals for Human Use

ICH M7

Application of the Principles of the ICH M7 Guidance to Calculation of Compound-Specific Acceptable Intakes; Application of the principles of the ICH M7 guideline to calculation of compound-specific acceptable intakes; principles applied to calculation of compound-specific AIs; application of principles to calculation of compound-specific acceptable intakes; Application of the principles of the ICH M7 guideline to calculation of compound-specific acceptable intakes.

ICH Q3A

impurities in new drug substances; Referenced regarding impurities and polymorphism.

ICH Q3B(R2)

Impurities in New Drug Products

ICH Q11

Development and Manufacture of Drug Substances; General principles for selection of starting materials

Related MFDS Guidelines

Korean regulatory guidelines covering similar topics

See Also (8)

Questions and Answers | M7(R2) Assessment and Control of DNA Reactive (Mutagenic) Impurities in Pharmaceuticals To Limit Potential Carcinogenic Risk: Guidance for Industry | Guideline Explorer | BioRegHub