This document discusses Regenerative Medicine Advanced Therapy (RMAT) designation, an expedited program for regenerative medicine therapies intended to treat serious or life-threatening diseases. To be eligible for RMAT, a therapy must be a cell or gene therapy, tissue product, or combination intended to treat a serious disease, and preliminary evidence must show potential to address unmet medical needs. A sponsor requests RMAT designation by submitting data to FDA describing the therapy, disease, available treatments, unmet need, and preliminary evidence of benefit. FDA will review the request and notify the sponsor within 60 days if RMAT is granted.

1. Regenerative Medicine
Advanced Therapy
(RMAT) Designation.
Reena Malik

2. Expedited Programs
• EXPEDITED PROGRAMS FOR REGENERATIVE MEDICINE THERAPIES
• Fast Track Designation
• Breakthrough Therapy Designation (BTD)
• Regenerative Medicine Advanced Therapy (RMAT Designation)
• Priority Review Designation
• Accelerated Approval

3. RMAT Eligibility
As described in Section
3033 of the 21st Century
Cures Act, a drug is
eligible for regenerative
medicine advanced
therapy (RMAT)
designation if:
The drug is a regenerative
medicine therapy, which is
defined as a cell therapy,
therapeutic tissue
engineering product, human
cell and tissue product, or
any combination product
using such therapies or
products, except for those
regulated solely under
Section 361 of the Public
Health Service Act and part
1271 of Title 21, Code of
Federal Regulations;
The drug is intended to
treat, modify, reverse, or
cure a serious or life-
threatening disease or
condition; and
Preliminary clinical
evidence indicates that
the drug has the
potential to address
unmet medical needs
for such disease or
condition.
Based on FDA’s
interpretation of Section
506(g) of the Food,
Drug, and Cosmetic Act
(as added by Section
3033 of the 21st Century
Cures Act), certain
human gene therapies
and xenogeneic cell
products may also meet
the definition of a
regenerative medicine
therapy.

4. When to file RMAT ?
The request for RMAT
designation must be made either
concurrently with submission of
an Investigational New Drug
application (IND)
As an amendment to an existing
IND. FDA will not grant a RMAT
designation if an IND is on hold or
is placed on hold during the
designation review.
You may submit a request for
RMAT designation to:
Food and Drug Administration
Center for Biologics Evaluation
and Research
Office of Tissues and Advanced
Therapies
Document Control Center
10903 New Hampshire Avenue
WO71, G112
Silver Spring, MD 20993-0002
OR

5. COVER LETTER
If the RMAT designation request is submitted to
your IND as an amendment, the cover letter
should specify that the submission contains
a REQUEST FOR REGENERATIVE MEDICINE
ADVANCED THERAPY DESIGNATION in bold,
uppercase letters.
If the request is submitted with an initial IND, the
cover letter should specify that the submission
contains both an INITIAL INVESTIGATIONAL NEW
DRUG SUBMISSION and REQUEST FOR
REGENERATIVE MEDICINE ADVANCED THERAPY
DESIGNATION in bold, upper case letters.

6. RMAT Doc
• In general, such a request should contain a concise summary of information that supports the RMAT designation,
including
• A description of the investigational product, including a rationale for the investigational new drug meeting the
definition of a regenerative medicine therapy;
• A discussion to support that the disease or condition, or the aspect of the disease or condition, that the product is
intended to treat is serious;
• A summary of the risks and benefits associated with the therapies, if any, currently available for this condition;
• A description of the unmet medical need that the product has the potential to address; and
• The preliminary clinical evidence that the product has the potential to address the specified unmet medical need for
this serious condition.
• A request for designation as an RMAT should describe the preliminary clinical evidence supporting designation. A
description of the preliminary clinical evidence should include, for example, the conditions for product administration,
outcome assessment, and patient monitoring; a description of the patients and their outcomes, including the number of
patients who have received the drug; and the design, conduct, and analyses of any clinical investigations.

7. Comparison of BTD and RMAT Designation

8. Review
Timeline
No later than 60 calendar days after receipt
of the designation request, the Office of
Tissues and Advanced Therapies (OTAT) will
notify the sponsor as to whether RMAT
designation has been granted.
If OTAT determines that the RMAT
designation request was incomplete or that
the drug development program does not
meet the criteria for RMAT designation,
OTAT will include a written description of
the rationale for such determination.

9. Reference
Guidance for Industry, Expedited Programs for
Regenerative Medicine Therapies for Serious
Conditions.
Cumulative CBER Regenerative Medicine
Advanced Therapy (RMAT) Designation
Requests Received by Fiscal Year
CBER Regenerative Medicine Advanced Therapy
(RMAT) Approvals
CBER Regenerative Medicine Advanced Therapy
(RMAT) Designations Withdrawn after Granting
or Rescinded by Fiscal Year