FDA Guidance Documents

Safety Assessment of Genome Editing Human Gene Therapy Products Using Next-Generation Sequencing

FDA's Center for Biologics Evaluation and Research has issued draft guidance recommending next-generation sequencing (NGS)-based methods for assessing safety of human genome editing gene therapy products in nonclinical studies. The guidance addresses evaluation of off-target editing risks and genome integrity for products supporting IND and BLA applications. Comments are being accepted via regulations.gov under docket FDA-2026-D-1255.

Bioanalytical Method Validation for Biomarker Concentrations Guidance

FDA's Center for Drug Evaluation and Research issued guidance FDA-2017-D-6821 to help sponsors validate bioanalytical methods used to evaluate biomarker concentrations in drug and biologic development programs. The guidance applies to sponsors of investigational new drug applications (INDs), new drug applications (NDAs), biologics license applications (BLAs), and abbreviated new drug applications (ANDAs). It addresses method validation for biomarker analysis in both clinical study samples and nonclinical study samples.

Guidance on Incorporating Voluntary Patient Preference Information

The FDA has issued updated guidance on incorporating voluntary patient preference information (PPI) into medical device submissions. This guidance provides recommendations on methods and contexts for collecting and submitting PPI to aid FDA decision-making throughout the total product lifecycle for biologics and medical devices.

FDA Guidance on Pyrogen and Endotoxin Testing

The FDA has issued guidance for biological product, drug, and device firms regarding pyrogen and endotoxin testing. This document provides recommendations based on current FDA thinking concerning USP and AAMI standards for bacterial endotoxin and pyrogen tests.

FDA Final Guidance on Topical Drug Product Characterization for ANDAs

The FDA has issued final guidance for industry on the physicochemical and structural characterization of topical drug products submitted in Abbreviated New Drug Applications (ANDAs). This guidance assists applicants in identifying generic topical products and supporting bioequivalence demonstrations. It finalizes the draft guidance issued in October 2022.

FDA Guidance on Medical Devices for Weight Loss

The FDA has issued guidance for medical devices intended for weight loss, outlining recommendations for non-clinical testing and clinical study design to support premarket submissions. This guidance aims to promote consistency and facilitate efficient review of submissions for devices related to weight loss, reduction, management, or obesity treatment.

FDA Q&A on Biosimilar Development and BPCI Act

The FDA has reissued its final guidance on biosimilar development and the BPCI Act, solely to withdraw three specific Q&As (I.8, I.10, and I.19). The agency is continuing to evaluate other Q&As for potential updates.

FDA Draft Q&As on Biosimilar Development and BPCI Act

The FDA has issued a draft guidance document providing updated answers to common questions regarding biosimilar development and the Biologics Price Competition and Innovation Act (BPCI Act). This revision replaces a previous draft and focuses on specific Q&As related to the abbreviated licensure pathway for biological products.

FDA Guidance: Flavored ENDS Premarket Applications

The FDA has issued guidance to assist with premarket tobacco product applications (PMTAs) for flavored electronic nicotine delivery systems (ENDS). This guidance, intended to improve submission and review efficiency, focuses on considerations related to youth risk. The document is non-binding and aims to inform potential future rulemaking.

Draft Guidance: Responding to FDA Form 483 Observations for Drug Manufacturers

The FDA has issued draft guidance for drug manufacturers on how to respond to observations noted on FDA Form 483 following CGMP inspections. This guidance is intended to assist domestic and foreign manufacturers in assessing conformity with CGMP requirements.