What is an RMAT and Who Has One? (30 Announced / 40 Granted)

Total RMATs Received and Awarded by FDA

To date, the U.S. FDA has received 108 total requests for RMAT designations and granted 40. This means that slightly over one-third of all RMAT applications get approved.

The table below presents the cumulative number of RMAT requests that the FDA has received by year.

*Source: U.S. FDA 

Regenerative Medicine Advanced Therapy (RMAT)

RMAT is an acronym that stands for “Regenerative Medicine Advanced Therapy”. Introduced in December 2016 as part of the 21st Century Cures Act, an RMAT designation can shorten time to market for innovative medicines.

An RMAT submission request must be made in conjunction with an Investigational New Drug application (IND), either concurrently or as an amendment to an existing IND.

Products eligible for an RMAT designation include:

• Cell therapies
• Gene therapies
• Therapeutic tissue engineering products
• Human cell and tissue products
• Combination products using such therapies or products

By definition, an RMAT is an award from the U.S. FDA that allows for faster, more streamlined approvals of regenerative medicine products within the United States, such as cell and gene therapies, tissue engineering products, and combination products. RMAT designations make innovative products eligible for quicker development and review of a marketing application.

An RMAT designation is similar to the breakthrough therapy designation that is available to drugs.

Benefits of an RMAT include increased opportunities to meet with FDA officials, as well as early meetings to discuss potential surrogate or intermediate endpoints.

21st Century Cures Act

The 21st Century Cures Act was passed by the 114th U.S. Congress in December 2016. The act authorize $6.3 billion in funding, most of which is allocated for the National Institutes of Health (NIH).

The bill, passed by both houses of Congress and signed into law by President Obama, puts the U.S. on the same playing field as other countries that support accelerated of innovative medicines. Other regions that have accelerated pathways include Japan, South Korea, and the EU. The bill encourages late-stage development in regenerative medicine, recognizing the field’s potential to address severe unmet medical needs.

Congress included several provisions related to cell and gene therapies in the 21st Century Cures Act, the most important of which is the RMAT designation.

Regenerative Medicine Advanced Therapy Reviews by The FDA