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Oligonucleotide Drug Products: Administrative and Procedural Recommendations Guidance for Sponsor-Investigators: Draft Guidance for Sponsor-Investigators

Description

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.

Scope & Applicability

Regulatory Context

• 21CFR312.3§ 312.3 Definitions and interpretations.

  (a) The definitions and interpretations of terms contained in section 201 of the Act apply to those terms when used in this part:Read full regulation →

• 21CFR312.81§ 312.81 Scope.

  This section applies to new drug and biological products that are being studied for their safety and effectiveness in treating life-threatening or severely-debilitating diseases.Read full regulation →

• 21CFR20.61§ 20.61 Trade secrets and commercial or financial information which is privileged or confidential.

  (a) A trade secret may consist of any commercially valuable plan, formula, process, or device that is used for the making, preparing, compounding, or processing of trade commodities and that can be said to be the end product of either innovation or substantial effort. There must be a direct relationRead full regulation →

• 21CFR20.21§ 20.21 Uniform access to records.

  Any record of the Food and Drug Administration that is disclosed in an authorized manner to any member of the public is available for disclosure to all members of the public, except that:Read full regulation →

• 21CFR312.23§ 312.23 IND content and format.

  (a) A sponsor who intends to conduct a clinical investigation subject to this part shall submit an “Investigational New Drug Application” (IND) including, in the following order:Read full regulation →

• 21CFR312.32§ 312.32 IND safety reporting.

  (a) Definitions. The following definitions of terms apply to this section:Read full regulation →

• 21CFR312.33§ 312.33 Annual reports.

  A sponsor shall within 60 days of the anniversary date that the IND went into effect, submit a brief report of the progress of the investigation that includes:Read full regulation →

• 21CFR56.111§ 56.111 Criteria for IRB approval of research.

  (a) In order to approve research covered by these regulations the IRB shall determine that all of the following requirements are satisfied:Read full regulation →

• 21CFR50.20§ 50.20 General requirements for informed consent.

  Except as provided in §§ 50.22 , 50.23 , and 50.24 , no investigator may involve a human being as a subject in research covered by these regulations unless the investigator has obtained the legally effective informed consent of the subject or the subject's legally authorized representative. An invesRead full regulation →

• Clinical Investigator

  Mark J. Savant, M.D

  2025-07-15

• Clinical Investigator

  Americo F. Padilla, M.D.

  2025-03-25

• Institutional Review Board (IRB)

  Armstrong County Memorial Hospital

  2024-11-05

• Clinical Investigator

  Namita A. Goyal, M.D.

  2024-10-22

• Institutional Review Board (IRB)

  Louisiana State University Health Science Center IRB

  2024-10-08

• Bioresearch Monitoring Program/IRB

  Massachusetts Institute of Technology MIT

  2024-07-16

• Clinical Investigator (Sponsor)

  Angela D. Ritter, M.D.

  2024-06-18

• Institutional Review Board (IRB)

  New York State Psychiatric Institute IRB

  2024-03-26

• Clinical Investigator

  Jeffrey W. Taub, M.D./Children's Hospital of Michigan

  2024-01-09

• Clinical Investigator

  Luis Javier Pena-Hernandez, M.D., FCCP

  2023-09-29

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