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Regulatory Focus™ > News Articles > 2020 > 7 > Update on RMAT designations

Update on RMAT designations

Posted 15 July 2020 | By William K. Sietsema, PhDJanet Lynch Lambert, MBA 

Update on RMAT designations

This article discusses the scope and purpose of the special designation for regenerative medicine advanced therapies (RMAT) created by the passage of the 21st Century Cures Act. The authors explain the benefits expected to be realized with RMAT and provide a tally of products receiving the special designation to date and a current count, by year, of products for which RMAT designation has been requested.
Section 3033 of the 21st Century Cures Act, titled “Accelerated Approval for Regenerative Advanced Therapies” created a special designation for regenerative medicine advanced therapies.
The regenerative medicine and advanced therapy field has the potential to provide profound, durable, and possibly curative benefits to patients with serious diseases and disorders – and the RMAT designation is helping drive these transformative technologies to market. In the 3 years since the creation of the RMAT designation, it has been effectively implemented by the US Food and Drug Administration (FDA), and more than 50 sponsors have been granted the designation for a range of technologies and indications. The Alliance for Regenerative Medicine and its member organizations were instrumental in working with the US Congress, FDA, and other stakeholders to create this new regulatory pathway for advanced therapies.
A product is eligible for RMAT designation if it is a regenerative medicine therapy, such as cell therapy, therapeutic tissue-engineering product, human cell and tissue product, gene therapy, or any combination product using such therapies or products. It is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition and preliminary clinical evidence indicates the drug has the potential to address unmet medical needs for such disease or condition.
Other national regulatory bodies have, in recent years, made special regulatory provisions to recognize the immense value of regenerative medicine products. For example, Japan’s SAKIGAKE designation was introduced in 2015 to promote research and development of regenerative medicines and other innovate pharmaceuticals and medical devices. However, the RMAT designation is unique both in the number of applicants and the number of designations granted. The RMAT designation has, therefore, helped keep the US competitive in the global field, while addressing specific needs and requirements of regenerative medicine advanced therapy products.
The RMAT designation provides largely the same benefits as breakthrough therapies and includes interactions with FDA to expedite development and review of the product and consideration of the product for priority review or accelerated approval. Receiving RMAT designation also allows for increased flexibility in clinical trial design, for instance, in the number of clinical trial sites. Developers can also use patient registry data and other sources of real-world data in postapproval pathways.
However, while developers seeking breakthrough designation are required to show that their therapeutic candidate would provide a substantial improvement over existing therapies, RMAT designation requires only that the therapy have the potential to address unmet medical need. This provides more flexibility to developers in this field and further drives innovation.

RMAT designations have been available now for 4 years, so what have we seen so far?
RMAT designations to date
Table 1 provides the FDA’s latest tally of RMAT designations by year. For 2017, the data represent about 9 months of activity, and for 2020, 6 months of activity. The number of requests received seems to have increased in 2020, considering that the 31 reported requests are from the first 6 months of the year. The number granted in the first half of 2020 has remained comparable with other years, despite the challenges of – and the bandwidth required by -- COVID-19. However, the effect of the COVID-19 pandemic on the amount of time for an RMAT designation request to be reviewed remains unknown.


So far, no RMAT-designated products have received marketing approval – although decisions on several product candidates with RMAT designation are expected in the near term. These decisions include Enzyvant’s RVT-802, for the treatment of pediatric congenital athymia; Bristol Meyers Squibb’s Liso-cel, for relapsed or refractory large B-cell lymphoma; and Mallinckrodt’s StrataGraft, for the treatment of severe burns. To date, no RMAT designations have been withdrawn or rescinded after being granted.
Requests by therapeutic area
Figure 1 provides metrics on designation requests by therapeutic area for RMAT designations through July 9, 2020. Neurology (33 requests) is the largest category, followed by oncology (hematology plus solid tumors; 28). The orthopedic (15), cardiovascular (13), and dermatology (12) categories are also well represented.


Requests by product type
Figure 2 provides metrics on designation requests by product type for RMAT designations through 9 July 2020. The greatest number of designation requests have been for cell therapy products (73 requests), a combination of allogeneic (43) and autologous (30). Gene therapy products (55) also account for a large proportion of these requests.


Publicly announced RMAT designations
Tables 2A, 2B, and 2C provide listings of the publicly announced RMAT designations. As of 30 June 2020, 43 designations had been announced and 52 were granted, thus 9 are currently unannounced.




Clearly, the RMAT designation program has been very active for the FDA and popular for sponsors. With Enzyvant, Mallinckrodt, and Bristol Myers Squibb working with the FDA to bring their respective product candidates to market, we are looking forward to seeing the first wave of approvals from this program.
  1. US Food and Drug Administration. Cumulative CBER regenerative medicine advanced therapy (RMAT) designation requests received by fiscal year.  Current as of 9 July 2020. Accessed 14 July 2020.   
  2. Alliance for Regenerative Medicine. Expedited approval designations. Accessed 14 July 2020.
  3. Bioinformant. RMAT designations. Last updated 12 May 2020. Accessed 14 July 2020.
  4. The Niche – Knoepfler Lab Stem Cell Blog. RMAT list (44). Last updated 10 July 2010. Accessed 14 July 2020.
About the authors
William K. Sietsema, PhD, is vice president, global regulatory affairs at Caladrius Biosciences, a company that focuses on innovative cell therapies for difficult-to-treat diseases. Before joining Caladrius, he was global regulatory lead at Amgen, where he provided strategic guidance to a portfolio of early stage projects in oncology and inflammation. He was also vice president, global regulatory consulting and submissions, at Kendle International/INC Research, and adjunct professor of pharmaceutical sciences at the University of Cincinnati, College of Pharmacy, where he taught classes in drug development. He has 35 years of experience in the pharmaceutical industry. He received his PhD in biochemistry from the University of Wisconsin, Madison, in 1982. He is a member of the American Chemical Society, the Association for Regenerative Medicine, and RAPS. He was recognized by R&D Directions as one of the top 20 clinical research scientists in 2007. He can be contacted at
Janet Lynch Lambert, MBA, joined the Alliance for Regenerative Medicine in 2017 as the organization’s first CEO. With more than 25 years in public and private sector management, Lambert is an experienced government relations and business professional with an extensive record of accomplishment. She most recently served as the acting head of engagement for the All of Us Research Program at the National Institutes of Health and as head of the Outreach Office in the Office of the NIH Director. Before joining NIH, she was vice president of government relations and head of the Washington office of Life Technologies, where she aided the company in its growth from $300 million in annual sales to more than $3 billion. Lambert received her MBA in international business from Georgetown University.
Citation Sietsema WK, Lambert JL. Regulatory Intelligence: Update on RMAT designations. Regulatory Focus. July 2020. Regulatory Affairs Professionals Society.

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