IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Clinical Recommendations: Draft Guidance for Industry

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 (SDLT) genetic disease. Most often, individuals with such diseases will have no alternative treatment options, and their diseases will be rapidly progressing, resulting in early death and/or devastating or irreversible morbidity within a short time frame without treatment. In these situations, drug development targeted to a larger number of patients with the same disease is not anticipated because of the specificity of the mechanism of action of the ASO combined with the rarity of the treatment-amenable patient population. The gene variant or variants that are targeted by the ASO drug product should be unique to the trial participant(s) and generally only reported in a small number of patients (typically 1 to 2) in the disease population. If more than a few patients may be candidates for targeted treatment with the ASO drug product, then the ASO is no longer considered individualized, and the sponsor should discuss a drug development plan of the investigational ASO drug product for a larger patient population with the relevant review division.

Scope & Applicability

Regulatory Context

• 21CFR50.25§ 50.25 Elements of informed consent.

  (a) Basic elements of informed consent. In seeking informed consent, the following information shall be provided to each subject: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 →

• 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 →

• 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 →

• 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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