To date, 44 RMAT (Regenerative Medicine Advanced Therapy) designations have been publicly announced. However, the FDA states it has received 139 requests and issued 48.

Sponsors of cell and gene therapies are eligible to obtain an RMAT designation from the U.S. FDA if their product is intended to treat serious or life-threatening diseases and there is preliminary clinical evidence that it can address unmet medical needs.

The most recent addition to this list was the RMAT jointly awarded to CRISPR Therapeutics and Vertex Pharmaceuticals for CTX001™ in the treatment of severe hemoglobinopathies.

Prior to that, it was Immunicum’s ilixadencel on May 6, 2020. Ilixadencel consists of activated allogeneic dendritic cells used as an immune primer for the treatment of metastatic Renal Cell Carcinoma (mRCC).

RMAT Designations by the U.S. FDA

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