The 21st Century Cures Act was groundbreaking and brought limitless possibilities to the field of medical treatment.  Regenerative medicine advanced therapy, or RMAT, designations were created under this act to provide ways for patients with life-threatening conditions to receive cutting-edge medical innovations. RMATs have wide scope of applications  because they focus on creating a living, functional tissue and using it in treatments. Most importantly, healthcare experts believe that it may significantly relieve the nationwide organ donation shortage. However, it is often very complicated for products to be RMAT designated. Since 2017, approximately 100 products have undergone the approval process but only about a third of them have been approved.  Also, it is important to note that being RMAT designated does not mean that the product has the approval to be marketed to the general population.

What is Regenerative Medicine?


The National Institute of Health (NIH) describes regenerative medicine as, “ the process of creating living, functional tissues to repair or replace tissue or organ function lost due to age, disease, damage, or congenital defect”.  It is a very effective treatment method because of its potential to treat incurable organ damage or conditions that endanger the lives of patients. In addition, scientists may grow organs and tissues in a laboratory to be used in implants. As a result, this procedure is expected to alleviate the shortage of organ donations and save numerous lives.


What does it mean to be RMAT designated


In December 2016, the 21st Century Cures Act was signed into law. It expedites the development of certain medical products and innovations, which will help  patients who need them the most.
Regenerative medicine advanced therapy (RMAT) designation was formulated specifically to provide patients with  therapeutic tissue engineered products, cell therapies, human cell and tissue products or any combination product that may save them from serious or life-threatening conditions or diseases which would otherwise,  be incurable.



RMAT designation requirements

Section 3033 of 21st Century Cures Act states that a drug is eligible for regenerative medicine advanced therapy (RMAT) designation if it meets these criteria:

     1. 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;

     2. The drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and

     3. Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such a disease or condition.

Companies should refer to Guidance for Industry Expedited Programs for Serious Conditions – Drugs and Biologics, section III. A. to check if the FDA deems a certain disease or condition to be a serious or life-threatening and whether a drug has the possibility of being RMAT designated to treat the condition.

To learn more about how to request a RMAT designation, please visit:

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

*As of March 31, 2019, only 33 products have been RMAT-designated and none of these RMAT designated products have received an approval to be marketed.

About RegDesk™

The RegDesk™ platform provides manufacturers of medical devices, as well as biotech companies, with the information on the most important changes in regulations across the world. Taking into account the importance of the compliance to the regulatory requirements, RegDesk™ creates the possibility to obtain reliable information.


Want to know more about our solutions? Speak to a RegDesk Expert today!