Regenerative Medicine Advanced Therapy (RMAT)- FDA explains which products fall under "RMAT"

Recommendation

9/10 May 2023
Heidelberg, Germany

Annex 2 & Co. - GMP Compliance for Biopharmaceuticals
Regulatory Requirements and Practical Implementation

The topic of regenerative medicine therapies for diseases with serious courses has been in the FDA's focus for some time. This has also become clear through guidelines such as "Expedited Programs for Regenerative Medicine Therapies for Serious Condition". But also through the specially prepared overviews of other relevant guidelines (Resources Related to Regenerative Medicine Therapies) in this context show this attention.

For a clearer explanation of when a product or therapy is a so-called "Regenerative Medicine Advanced Therapy", the FDA has now explained this term in accordance with Section 3033 of the 21st Century Cures Act and outlined the prerequisites for this classification:

• "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 conditio
• Based on FDA’s interpretation of Section 506(g) of the Federal 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."

A corresponding application for the designation as "Regenerative Medicine Advanced Therapy" can already be submitted with the marketing authorisation submission of an IND or can be submitted as an addition to an already existing IND.

Further details can be found directly under "Regenerative Medicine Advanced Therapy Designation".