Mesoblast Limited has announced that the United States Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for its novel mesenchymal precursor cell (MPC) therapy in the treatment of heart failure patients with left ventricular systolic dysfunction and left ventricular assist devices (LVADs). The RMAT designation under the 21st Century Cures Act aims to expedite the development of regenerative medicine therapies intended for the treatment of serious diseases and life-threatening conditions.
This RMAT designation allows for multi-disciplinary, comprehensive interactions with the FDA to support the efficient development of and potential accelerated approval pathway for Mesoblast’s allogeneic MPCs in the treatment of heart failure patients with LVADs. The RMAT designation also offers eligibility for priority review. Once the biologics license application (BLA) for a product is approved, the FDA can require various post-approval confirmatory commitments.
Mesoblast chief executive Silviu Itescu stated, “The RMAT designation speaks to the strength of the clinical data generated to date using our cell-based therapy in these heart failure patients with LVADs who are at risk of high mortality and have extremely limited treatment options. We are looking forward to working closely with the FDA in advancing this programme with the aim of providing a new therapeutic option for these patients with exceptionally high unmet clinical need.”
The basis of this RMAT designation grant came from the completed study data set of a 30-patient randomized, blinded, placebo-controlled pilot trial of Mesoblast’s MPCs at a dose of 25 million cells in heart failure patients with LVADs, and related analyses. These preliminary clinical data suggest that Mesoblast’s MPC product: improved native heart function, prolonged the time post LVAD implantation of a first hospitalization for a non-surgical major gastrointestinal (GI) bleeding event and improved early survival rates in these LVAD recipients.
The results of the pilot study were published in the American Heart Association Journal Circulation and can be found here. A phase 2b trial of MPCs at a dose of 150 million cells is currently being conducted in 159 patients with heart failure and LVADs and is funded by the NIH and the Canadian Institute of Health Research. This trial has completed enrollment and the primary endpoint will be reached in Q1 CY 2018. FDA has invited Mesoblast to have a multidisciplinary comprehensive discussion as soon as possible regarding the development strategy and the evidence needed to achieve an approval in an efficient manner.