Mesoblast has filed an application with the US Food and Drug Administration (FDA) seeking orphan drug and rare paediatric disease designations for its allogeneic cell therapy, Revascor (rexlemestrocel-L), to treat congenital heart disease hypoplastic left heart syndrome (HLHS).
The company has filed the applications based on Revascor’s data from a randomised, blinded, controlled prospective study carried out at a single centre in the US involving 19 children with HLHS.
HLHS is a severe congenital heart disease where the left side of the heart does not fully develop, leading to a decrease in efficient pumping of oxygenated blood from the left ventricle to the rest of the body.
Revascor is an allogeneic preparation of immunoselected and culture-expanded mesenchymal precursor cells.
These cells were earlier demonstrated to have multiple mechanisms of actions that may be beneficial to children with HLHS, including anti-fibrosis, anti-apoptosis, neovascularisation, immunomodulation, reduction in inflammation, and reversal of endothelial dysfunction.
A single intramyocardial administration of Revascor into the left ventricle resulted in significant improvement in LV ejection fraction at 12 months in the DREAM-HF randomised sham-placebo controlled prospective trial of the therapy in 565 adults with heart failure with low ejection fraction (HFrEF).
In the HLHS study, a single intramyocardial Revascor administration during staged surgery resulted in an increase in LV systolic and diastolic volumes over 12 months, compared to the control group.
Mesoblast is focused on the development of allogeneic (off-the-shelf) cellular medicines to treat severe and life-threatening inflammatory conditions.