U.S. flag An official website of the United States government

U.S. Food and Drug Administration
  1. Home
  2. Regulatory Information
  3. Search for FDA Guidance Documents
  4. Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations, Guidance for Sponsor-Investigators
  1. Search for FDA Guidance Documents

GUIDANCE DOCUMENT

Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations, Guidance for Sponsor-Investigators Draft Guidance for Industry December 2021

Download the Draft Guidance Document Read the Federal Register Notice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Not for implementation.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-2021-D-1139
Issued by:
Guidance Issuing Office
Center for Drug Evaluation and Research

The purpose of this guidance is to provide recommendations regarding the chemistry, manufacturing, and controls (CMC) information that should be provided in an investigational new drug application (IND) submitted by a sponsor-investigator (hereafter referred to as sponsor) developing an individualized antisense oligonucleotide (ASO) drug product for a severely debilitating or life-threatening (SDLT) disease caused by a unique genetic variant where only a small number of individuals are prospectively identified (typically one or two). These individualized ASO drug products should be from a well-characterized chemical class for which there is substantial clinical and nonclinical experience that is either publicly available or to which the sponsor has a right to reference.

Submit Comments

Submit Comments Online

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-2021-D-1139.

Back to Top