FDA Grants Revascor® (Rexlemestrocel-L) Regenerative Medicine Advanced Therapy (RMAT) Designation in Children with Congenital Heart Disease

Core Insights - Mesoblast Limited has received FDA's Regenerative Medicine Advanced Therapy (RMAT) designation for its stromal cell therapy Revascor® (rexlemestrocel-L) aimed at treating hypoplastic left heart syndrome (HLHS) in children, following positive results from a randomized controlled trial [1][2][3] Group 1: FDA Designations and Implications - The RMAT designation allows Mesoblast to expedite the development of Revascor, providing benefits such as rolling review and eligibility for priority review upon filing a Biologics License Application (BLA) [3] - Earlier in the year, Revascor was also granted Rare Pediatric Disease Designation (RPDD) and Orphan-Drug Designation (ODD) by the FDA, indicating the serious nature of HLHS and its rarity [2] Group 2: Clinical Trial Results - A blinded, randomized, placebo-controlled trial involving 19 children with HLHS demonstrated that a single administration of Revascor led to significantly larger increases in left ventricular end-systolic and end-diastolic volumes over 12 months compared to controls, with p-values of 0.009 and 0.020 respectively [4] - These results suggest clinically important growth of the left ventricle, which is crucial for successful surgical correction and reducing the risk of heart failure and other complications [5] Group 3: About HLHS and Revascor - HLHS is a severe congenital heart defect that can lead to high neonatal mortality rates, accounting for 25% to 40% of all neonatal cardiac deaths if not treated promptly [6] - Revascor is an allogeneic therapy derived from immunoselected mesenchymal precursor cells, which may provide multiple benefits including neovascularization and reduction of inflammation [7] Group 4: Company Overview - Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory conditions, with a strong intellectual property portfolio extending to at least 2041 [8][9] - The company is also advancing other product candidates for various indications, including remestemcel-L for inflammatory diseases and rexlemestrocel-L for chronic heart failure [10]