Issue Date: June 14, 2024
Issued by: Center for Drug Evaluation and Research
Topic(s): Clinical – Pharmacology
Status: Final
Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics: Guidance for Industry
This guidance provides recommendations to assist industry in the development of oligonucleotide therapeutics under section 505 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355) and 21 CFR parts 312 and 314. Specifically, this guidance represents the FDA’s recommendations for certain evaluations during development of oligonucleotide therapeutics, including: (1) characterizing the potential for QTc interval prolongation, (2) performing immunogenicity risk assessment, (3) characterizing the impact of hepatic and renal impairment, and (4) assessing the potential for drug-drug interactions. This guidance provides recommendations on when to conduct these assessments and what types of assessments are suitable to address the topics listed above. Additional Guidance Resources: Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics: Guidance Snapshot Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics: Guidance Recap Podcast Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics Guidance Recap Podcast Transcript
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