Mesoblast has submitted applications for orphan drug and pediatric rare disease designations for rexlemestrocel-L, also known as Revascor. The applications are based on data from a controlled, randomized, and blinded study conducted at a single U.S. center, involving 19 children diagnosed with Hypoplastic Left Heart Syndrome (HLHS).
HLHS is a serious congenital heart disease characterized by underdevelopment of the left side of the heart. This condition hinders the efficient pumping of oxygenated blood from the left ventricle to the rest of the body.
Advancements in Pediatric Heart Treatment and Adult Heart Failure
Revascor is an allogeneic preparation of culture-expanded and immunoselected mesenchymal precursor cells. These cells have been previously shown to possess multiple beneficial mechanisms of action for children with HLHS. These include anti-apoptosis, anti-fibrosis, neovascularisation, immunomodulation, reduction in inflammation, and the reversal of endothelial dysfunction.
A single intramyocardial administration of Revascor into the left ventricle resulted in a significant improvement in the Left Ventricle (LV) ejection fraction at 12 months. This result was observed in the DREAM-HF randomized sham-placebo-controlled prospective trial of the therapy in 565 adults diagnosed with heart failure with low ejection fraction (HFrEF).
Mesoblast’s Rexlemestrocel-L Shows Promise for Pediatric Heart Treatment
In the HLHS study, a single intramyocardial administration of Revascor during staged surgical procedures led to an increase in LV systolic and diastolic volumes over 12 months, compared to the control group.
Mesoblast is primarily focused on the development of allogeneic (off-the-shelf) cellular medicines. The company aims to address severe and life-threatening inflammatory conditions.
The applications for rexlemestrocel-L are based on promising data from a study involving children with HLHS. The study demonstrated the beneficial effects of a single intramyocardial administration of Revascor, including significant improvements in LV ejection fraction and LV systolic and diastolic volumes. Mesoblast’s commitment to developing effective treatments for serious and life-threatening conditions is further emphasized with these applications.
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