Pharmacogenomic Data Submissions: Guidance for Industry

Description

This guidance is intended to facilitate scientific progress in the field of pharmacogenomics and to facilitate the use of pharmacogenomic data in drug development. The guidance provides recommendations to sponsors holding investigational new drug applications (INDs), new drug applications (NDAs), and biologics license applications (BLAs) on (1) when to submit pharmacogenomic data to the Agency during the drug or biological drug product2 development and review processes, (2) what format and content to provide for submissions, and (3) how and when the data will be used in regulatory decision making. Key information, including examples of when pharmacogenomic data submissions would be required and when voluntary genomic data submissions (VGDSs) would be welcome are provided in a separate companion document (Pharmacogenomic Data Submissions, Attachment: Examples of Voluntary Submissions or Submissions Required Under 21 CFR 312, 314, or 601).

Scope & Applicability

Regulatory Context

• 21CFR601.12§ 601.12 Changes to an approved application.

  (a) General.Read full regulation →

• 21CFR312.22§ 312.22 General principles of the IND submission.

  (a) FDA's primary objectives in reviewing an IND are, in all phases of the investigation, to assure the safety and rights of subjects, and, in Phase 2 and 3, to help assure that the quality of the scientific evaluation of drugs is adequate to permit an evaluation of the drug's effectiveness and safeRead 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 →

• 21CFR314.50§ 314.50 Content and format of an NDA.

  NDAs and supplements to approved NDAs are required to be submitted in the form and contain the information, as appropriate for the particular submission, required under this section. Three copies of the NDA are required: An archival copy, a review copy, and a field copy. An NDA for a new chemical enRead full regulation →

• 21CFR601.2§ 601.2 Applications for biologics licenses; procedures for filing.

  (a) General. To obtain a biologics license under section 351 of the Public Health Service Act for any biological product, the manufacturer shall submit an application to the Director, Center for Biologics Evaluation and Research or the Director, Center for Drug Evaluation and Research (see mailing aRead full regulation →

• 21CFR312.30§ 312.30 Protocol amendments.

  Once an IND is in effect, a sponsor shall amend it as needed to ensure that the clinical investigations are conducted according to protocols included in the application. This section sets forth the provisions under which new protocols may be submitted and changes in previously submitted protocols maRead full regulation →

• 21CFR312.31§ 312.31 Information amendments.

  (a) Requirement for information amendment. A sponsor shall report in an information amendment essential information on the IND that is not within the scope of a protocol amendment, IND safety reports, or annual report. Examples of information requiring an information amendment include:Read full regulation →

• 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 →

• 21CFR314.81§ 314.81 Other postmarketing reports.

  (a) Applicability. Each applicant shall make the reports for each of its approved applications and abbreviated applications required under this section and section 505(k) of the act.Read full regulation →

• In Vivo Bioavailability-Bioequivalence Studies – Clinical

  Maria A. Carballosa, M.D.

  2025-12-23

• Unapproved New Drug/Unlicensed Biological Product/Biologics License Application (BLA)

  Celularity, Inc

  2025-12-09

• Sponsor/Investigator

  Verdure Sciences, Inc.

  2025-11-18

• Clinical Investigator (Sponsor)

  Pamela K. Den Besten, DDS, MS

  2025-09-30

• Clinical Investigator/Sponsor

  Ralph A. DeFronzo, M.D.

  2025-09-23

• False & Misleading Claims/Misbranded

  Novo Nordisk Inc.

  2025-09-16

• False & Misleading Claims/Misbranded

  Eli Lilly and Company

  2025-09-16

• Clinical Investigator

  Shirish M. Gadgeel, M.D.

  2025-09-09

• Good Laboratory Practice (GLP) for Nonclinical Laboratory Studies

  CCIC Huatongwei International Inspection Co., Ltd.

  2025-08-26

• Good Laboratory Practice (GLP) for Nonclinical Laboratory Studies

  Jiangsu Kerbio Medical Technology Group Co.

  2025-08-26

• Evidentiary Expectations for 510(k) Implant Devices: Draft Guidance for Industry and Food and Drug Administration Staff (Status: Draft)
• Redbook 2000: IV.C.9.b. Guidelines for Developmental Toxicity Studies (Status: Final)
• Redbook 2000: IV.C.9.a.Guidelines for Reproduction Studies (Status: Final)
• Redbook 2000: IV.C.5.b. One-Year Toxicity Studies with Non-Rodents (Status: Final)
• Redbook 2000: IV.C.4.b. Subchronic Toxicity Studies with Non-Rodents (Status: Final)
• Redbook 2000: IV.C.4.a. Subchronic Toxicity Studies with Rodents (Status: Final)
• Redbook 2000: IV.C.3.b. Short-Term Toxicity Studies with Non-Rodents (Status: Final)
• Redbook 2000: IV.C.3.a. Short-Term Toxicity Studies with Rodents (Status: Final)