Surge in RMAT designations awarded by the FDA

Since the first time the US Food and Drug Administration (FDA) awarded a regenerative medicine advanced therapeutic (RMAT) designation in 2017, activity in this space remained relatively modest until 2023.

The number of RMAT designations awarded by the FDA increased sharply from 2024, reaching a record of 48 RMAT designations in 2025. This rapid growth reflects the expanding regenerative medicine landscape and suggests that a larger number of these therapies are progressing towards regulatory review.

In December 2016, the RMAT designation was signed into US law and was designed to expedite the development and review of regenerative medicine therapies that are intended to treat, modify, reverse, or cure a serious condition. To qualify for RMAT designation, a product must be supported by preliminary clinical evidence demonstrating its potential to address unmet medical needs.

In 2017, the FDA awarded 15 RMAT designations. From 2017–23, the number of RMAT designations remained relatively low, averaging 13.9 per year. However, from 2024, the rates of RMATs awarded by the FDA rose substantially (Figure 1).

A total of 43 RMAT designations were awarded in 2024, followed by a peak of 48 RMAT designations in 2025, at an almost three-fold increase from the 17 designations awarded in 2023. Collectively, the 91 RMAT designations awarded in 2024 and 2025 accounted for 48% of all RMAT designations awarded since the program began, highlighting the significant recent acceleration in FDA RMAT designation activity.

The surge in RMAT designations awarded from 2024 coincides with the maturation of the cell and gene therapy (CGT) pipeline. CGTs represent a key component of the regenerative medicine landscape, as these therapies use engineered cells or genetic modification to restore, replace, or modify biological function. Currently, 2,315 CGT products are in Phases I, II, or III of clinical development. This is the highest number of CGTs recorded, with a compound annual growth rate of 7.35% since 2020. As this expanding pipeline progresses through clinical development, an increasing number of regenerative medicines are generating the preliminary clinical evidence required for RMAT designation eligibility. Consequently, the pool of qualifying therapies has increased, contributing to the marked rise in RMAT designations observed in recent years. Conversely, the introduction of the RMAT designation may itself have supported growth in the CGT pipeline by providing developers with an expedited regulatory pathway, encouraging investment in and clinical development of regenerative medicine products.

Of the 73 individual drugs that received at least one RMAT designation in 2024 or 2025, Cabaletta Bio’s resecabtagene autoleucel received four RMAT designations, which is the highest number awarded to any product during this period. This chimeric antigen receptor (CAR) T cell therapy is designed to restore immune homeostasis and demonstrates the increasing maturity of regenerative medicine products entering later-stage clinical development, with ongoing Phase II and III trials across multiple autoimmune indications.

Driven by the expanding clinical-stage regenerative medicine pipeline, the RMAT program has entered a period of accelerated growth. This trend suggests growing momentum in regenerative medicine development, which may result in increased regulatory review and activity in upcoming years.