Regenerative medicine therapies (RMTs) are defined in section 506(g)(8) of the Food Drug & Cosmetic Act as including cell therapies, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies or products, except for those regulated solely under section 361 of the Public Health Service Act (42 U.S.C. 264) and Title 21 of the Code of Federal Regulations Part 1271 (21 CFR Part 1271).

Based on FDA’s interpretation of section 506(g), human gene therapy products, including genetically modified cells that lead to a sustained effect on cells or tissues, and xenogeneic cell products may meet the definition of an RMT. Further, for the purposes of section 506(g)(8), FDA interprets cell therapies to include both allogeneic and autologous cell therapies. Microorganisms (e.g., viruses, bacteria, fungi) that are not genetically modified do not meet the definition of regenerative medicine therapy.

Section 3033 of the 21st Century Cures Act established the Regenerative Medicine Advanced Therapy Designation (RMAT) to facilitate the development and review of certain RMTs. Additional information on RMTs and the RMAT designation can be found at the websites listed below.

Information on FDA’s Framework for the Regulation of Regenerative Medicine Products

• https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/framework-regulation-regenerative-medicine-products
• https://www.fda.gov/news-events/press-announcements/fda-announces-comprehensive-regenerative-medicine-policy-framework
• https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-fdas-comprehensive-new-policy-approach-facilitating

Overview of FDA’s Regenerative Medicine Advanced Therapy Designation

• https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation

Cellular & Gene Therapy Guidances

Guidances related to the development of regenerative medicine therapies

• https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/default.htm

CBER Standards Development Activities Related to Regenerative Medicine

• https://www.fda.gov/vaccines-blood-biologics/standards-development-regenerative-medicine-therapies

CBER Workshops, Meetings, & Conferences

• https://www.fda.gov/BiologicsBloodVaccines/NewsEvents/WorkshopsMeetingsConferences/default.htm

Tissue & Advanced Therapies Research

CBER Research Related to Regenerative Medicine

• https://www.fda.gov/vaccines-blood-biologics/biologics-research-projects/tissue-advanced-therapies-research

CBER Regenerative Medicine Fellowship

• https://www.fda.gov/vaccines-blood-biologics/regenerative-medicine-fellowship-under-service-fellowship-program-cber

Statement from FDA Commissioner Scott Gottlieb, M.D. on the FDA’s new policy steps and enforcement efforts to ensure proper oversight of stem cell therapies and regenerative medicine

• https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-fdas-new-policy-steps-and-enforcement-efforts-ensure

Webcast recording: Are Stem Cells Ready for Prime Time? A Look at FDA Research Advances in Regenerative Medicine

• https://www.fda.gov/science-research/about-science-research-fda/are-stem-cells-ready-prime-time-look-fda-research-advances-regenerative-medicine

Consumer Update: FDA Warns About Stem Cell Therapies

• https://www.fda.gov/consumers/consumer-updates/fda-warns-about-stem-cell-therapies

Remarks by Commissioner Gottlieb to the Alliance for Regenerative Medicine's Annual Board Meeting

• https://www.fda.gov/news-events/speeches-fda-officials/remarks-alliance-regenerative-medicines-annual-board-meeting-05222018

CBER Regenerative Medicine Advanced Therapy Designation Requests

• https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/cumulative-cber-regenerative-medicine-advanced-therapy-rmat-designation-requests-received-fiscal