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Nonclinical Testing Draft Guidance

Nonclinical Testing of Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases Guidance for Sponsor-Investigators

Draft Guidance for Sponsor-Investigators

April 2021

Not for implementation. Contains non-binding recommendations.

Docket Number: FDA-2021-D-0320

Issued by: Center for Drug Evaluation and Research

The purpose of this guidance is to describe the nonclinical information that FDA recommends to support an investigational new drug application (IND) for an antisense oligonucleotide being developed to treat a severely debilitating or life-threatening (SDLT) disease caused by a unique genetic variant where only a small number of individuals are prospectively identified (usually one or two). The investigational antisense oligonucleotide should be from a well-characterized chemical class for which there is substantial nonclinical information and clinical experience that is publicly available or to which the sponsor-investigator (hereafter referred to as sponsor) has a right of reference.



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