On July 2nd, 2018, Cellerant Therapeutics was awarded a Regenerative Medicine Advanced Therapy (RMAT) designation for romyelocel-L (human myeloid progenitor cells) by the U.S. FDA. The treatment is used for the “prevention of serious bacterial and fungal infections in patients with de novo acute myeloid leukemia (AML) undergoing induction chemotherapy.”
Cellerant RMAT Designation
Romyelocel- is a universal, off-the-shelf cell therapy, because it does not require HLA matching between patient and donor. Neutropenia is a major side effect of myelosuppressive chemotherapies, including induction therapy. This can endanger patients by placing them at risk for life-threatening infections.
According to Ram Mandalam, President and CEO of Cellerant, “Receiving RMAT designation for romyelocel-L is an important milestone and recognition by the FDA of the potential of romyelocel-L to serve this unmet medical need.”
What is an RMAT?
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. RMAT stands for “Regenerative Medicine Advanced Therapy” designation.
An RMAT is defined as an award by the U.S. FDA that allows for faster, more streamlined approvals of regenerative medicine products within the U.S., including 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.
Who Else Has Been Awarded an RMAT?
To date, the U.S. FDA has awarded 20 total RMAT designations. 12 of these were awarded during 2017 and eight more (including the one to Cellerant) have been awarded from January 1 to July 2, 2018. Abeona Therapeutics has been the only company so far to be awarded two RMAT designations, which it received for EB-101 and ABO-102.