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Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases Guidance for Sponsor-Investigators Draft Guidance for Sponsor-Investigators April 2021
Not for implementation.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:
- Issued by:
Guidance Issuing OfficeCenter 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.
You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))
If unable to submit comments online, please mail written comments to:
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852
All written comments should be identified with this document's docket number: FDA-2021-D-0320.