Description
This guidance provides sponsors engaged in the development of regenerative medicine therapies for serious or life-threatening diseases or conditions 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). 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 the Center for Biologics Evaluation and Research (CBER) review staff. As a general matter, this guidance addresses regenerative medicine therapies regulated by CBER as biological products under the FD&C Act, section 351 of the Public Health Service Act (PHS Act) (42 U.S.C. 262), and applicable regulations.
Scope & Applicability
Product Classes
6Defined 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 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 allogeneic and autologous cell therapies
Includes genetically modified cells
May meet the definition of a regenerative medicine therapy
Products regulated by CBER as biological products; Subject of the expedited programs guidance
Stakeholders
1Entity responsible for submitting applications under section 524B
Regulatory Context
Attributes
4Measurement of potency for biological products
Designation for regenerative medicine advanced therapies
Status of an IND that affects RMAT review
specifications for the purity, strength, and composition of dietary supplements
Related CFR Sections (2)
- 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 →
- 21CFR312.120§ 312.120 Foreign clinical studies not conducted under an IND.
(a) Acceptance of studies.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)