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Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations, Guidance for Sponsor-Investigators : Draft Guidance for Industry

Description

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.

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 →

• 21CFR312.6§ 312.6 Labeling of an investigational new drug.

  (a) The immediate package of an investigational new drug intended for human use shall bear a label with the statement “Caution: New Drug—Limited by Federal (or United States) law to investigational use.”Read full regulation →

• 21CFR25.31§ 25.31 Human drugs and biologics.

  The classes of actions listed in this section are categorically excluded and, therefore, ordinarily do not require the preparation of an EA or an EIS: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 →

• 21CFR312.80§ 312.80 Purpose.

  The purpose of this section is to establish procedures designed to expedite the development, evaluation, and marketing of new therapies intended to treat persons with life-threatening and severely-debilitating illnesses, especially where no satisfactory alternative therapy exists. As stated § 314.10Read 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 →

• 21CFR25.30§ 25.30 General.

  The classes of actions listed in this section and §§ 25.31 through 25.35 are categorically excluded and, therefore, ordinarily do not require the preparation of an EA or an EIS:Read full regulation →

• 21CFR25.40§ 25.40 Environmental assessments.

  (a) As defined by CEQ in 40 CFR 1508.9 , an EA is a concise public document that serves to provide sufficient evidence and analysis for an agency to determine whether to prepare an EIS or a FONSI. The EA shall include brief discussions of the need for the proposal, of alternatives as required by secRead full regulation →

• 21CFR210.2§ 210.2 Applicability of current good manufacturing practice regulations.

  (a) The regulations in this part and in parts 211 , 213 , 225 , and 226 of this chapter as they may pertain to a drug; in parts 600 through 680 of this chapter as they may pertain to a biological product for human use; and in part 1271 of this chapter as they are applicable to a human cell, tissue, Read full regulation →

• In Vivo Bioavailability-Bioequivalence Studies – Clinical

  Maria A. Carballosa, M.D.

  2025-12-23

• CGMP/Finished Pharmaceuticals/Adulterated

  SV Labs Corporation

  2025-12-23

• CGMP/Finished Pharmaceuticals/Adulterated

  Guangdong Renhe Guozhuang Biotechnology Co., Ltd.

  2025-12-23

• CGMP/Finished Pharmaceuticals/Adulterated

  Medinatura New Mexico, Inc.

  2025-12-23

• CGMP/Finished Pharmaceuticals/Adulterated

  Sklar Personal Care Inc.

  2025-12-16

• CGMP/Deviations/Biologics License Application (BLA)

  Microvascular Tissue, Inc.

  2025-12-16

• CGMP/Finished Pharmaceuticals/Adulterated

  Catalent Indiana, LLC

  2025-12-11

• CGMP/Finished Pharmaceuticals/Adulterated

  DeVere Manufacturing Inc.

  2025-12-09

• CGMP/Finished Pharmaceuticals/Adulterated

  CDL Services, Inc. DBA Technichem

  2025-12-09

• CGMP/Finished Pharmaceuticals/Adulterated

  Seaway Pharma Inc.

  2025-12-09

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