Description
This guidance provides sponsors engaged in the development of regenerative medicine therapies for serious or life-threatening diseases or conditions1 with our recommendations on the expedited development and review of these therapies, including as provided under section 506(g) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), as added by section 3033 of the 21st Century Cures Act (Cures Act).2 Under section 506(g) of the FD&C Act, a regenerative medicine therapy can be designated as a regenerative advanced therapy if it meets certain criteria. FDA refers to such designation as “regenerative medicine advanced therapy” (RMAT) designation. (See section III.C of this document). This guidance describes the expedited programs available to sponsors of regenerative medicine therapies for serious conditions, including those products designated as RMATs. To that end, the guidance provides information about the provisions in the Cures Act regarding the use of the accelerated approval pathway for regenerative medicine therapies that have been granted designation as an RMAT. Finally, the guidance describes considerations in the clinical development of regenerative medicine therapies and opportunities for sponsors of such products to interact with CBER review staff.
Scope & Applicability
Product Classes
7Defined in section 506(g)(8) of the FD&C Act; Product category eligible for RMAT designation; Products granted RMAT designation and approved via accelerated approval
Products explicitly excluded from the scope of this guidance
Products combining drug, device, or biological constituents; Generally recommended for Enhanced Documentation; Requires 14971-based framework incorporating ICH Q9; A drug-device combination where the device constituent part detects ingestion.
Includes genetically modified cells
Scope of the standards recognition program
Statutory authorities applied to these products
GT products within the scope of this guidance include products that mediate their effect by the expression of transferred genetic materials.; The main subject of the S12 guidance document
Stakeholders
1Entity responsible for submitting applications under section 524B
Regulatory Context
Attributes
1Single mode of action expected to make the greatest contribution to therapeutic effects
Related CFR Sections (3)
- 21CFR3.2§ 3.2 Definitions.
For the purpose of this part:Read full regulation →
- 21CFR601.41§ 601.41 Approval based on a surrogate endpoint or on an effect on a clinical endpoint other than survival or irreversible morbidity.
FDA may grant marketing approval for a biological product on the basis of adequate and well-controlled clinical trials establishing that the biological product has an effect on a surrogate endpoint that is reasonably likely, based on epidemiologic, therapeutic, pathophysiologic, or other evidence, tRead full regulation →
- 21CFR601.43§ 601.43 Withdrawal procedures.
(a) For biological products approved under § 601.41 or § 601.42 , FDA may withdraw approval, following a hearing as provided in part 15 of this chapter , as modified by this section, if:Read full regulation →
Related Warning Letters (10)
- 2025-12-23
In Vivo Bioavailability-Bioequivalence Studies – Clinical
Maria A. Carballosa, M.D.
- 2025-12-16
CGMP/Deviations/Biologics License Application (BLA)
Microvascular Tissue, Inc.
- 2025-12-16
Deviations/CFR/Regulations for Human Cells, Tissues & Cellular Products (HCT/Ps)
Green Valley Fertility Partners
- 2025-12-09
Unapproved New Drug/Unlicensed Biological Product/Biologics License Application (BLA)
BioXtek LLC
- 2025-12-09
Unapproved New Drug/Unlicensed Biological Product/Biologics License Application (BLA)
Celularity, Inc
- 2025-12-09
Unapproved New Drug/Unlicensed Biological Product/Biologics License Application (BLA)
Lux Therapeutics LLC dba Ponya Therapeutics LLC
- 2025-11-18
Sponsor/Investigator
Verdure Sciences, Inc.
- 2025-10-07
CGMP/Deviations/Biologics License Application (BLA)
New Life Medical Services, LLC
- 2025-09-30
Clinical Investigator (Sponsor)
Pamela K. Den Besten, DDS, MS
- 2025-09-23
Clinical Investigator/Sponsor
Ralph A. DeFronzo, M.D.
See Also (8)
- Classification of Products as Drugs and Devices and Additional Product Classification Issues: Guidance for Industry and FDA Staff (Status: Final)
- Validation of Procedures for Processing of Human Tissues Intended for Transplantation: Guidance for Industry (Status: Final)
- Recommendations for Obtaining a Labeling Claim for Communicable Disease Donor Screening Tests Using Cadaveric Blood Specimens from Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps): Guidance for Industry (Status: Final)
- Assessing Donor Suitability and Blood and Blood Product Safety in Cases of Known or Suspected West Nile Virus Infection: Guidance for Industry (Status: Final)
- Compliance with 21 CFR Part 1271.150(c)(1) – Manufacturing Arrangements: Guidance for Industry (Status: Final)
- Eligibility Determination for Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products: Guidance for Industry (Status: Final)
- Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Somatic Cell Therapy Investigational New Drug Applications (INDs): Guidance for FDA Reviewers and Sponsors (Status: Final)
- Providing Regulatory Submissions in Electronic Format – Drug Establishment Registration and Drug Listing (Status: Final)