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GUIDANCE DOCUMENT

IND Submissions for Individualized Antisense Oligonucleotide Drug Products: Administrative and Procedural Recommendations Guidance for Sponsor-Investigators Draft Guidance for Sponsor-Investigators January 2021

Download the Draft Guidance Document Read the Federal Register Notice
Draft Level 1 Guidance

Not for implementation. Contains non-binding recommendations.

Docket Number:
FDA-2020-D-2199
Issued by:
Guidance Issuing Office
Center for Drug Evaluation and Research

This guidance is intended for sponsor-investigators (hereafter referred to as sponsors) developing individualized investigational antisense oligonucleotide (ASO) drug products for a severely debilitating or life-threatening genetic disease. Most often, individuals with such diseases will not have FDA-approved treatment options and their diseases will be rapidly progressing, resulting in early death and/or devastating irreversible morbidity within a short time frame. In these situations, drug development targeted at a larger number of patients with the ASO is not anticipated because of the specificity of the mechanism of action of the ASO drug product combined with the rarity of the treatment-amenable patient population.

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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:

Dockets Management
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-2020-D-2199.

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