Mesoblast has filed for orphan drug designation (ODD) and rare pediatric disease designation (RPDD) with the US Food and Drug Administration (FDA) for Revascor (rexlemestrocel-L) to treat congenital heart disease hypoplastic left heart syndrome (HLHS).
Revascor is an allogeneic cell therapy made from immunoselected and culture-expanded mesenchymal precursor cells.
Based in Australia, Mesoblast is engaged in the development of allogeneic cellular medicines for inflammatory diseases.
The filings were based on the findings from a blinded, randomised, controlled prospective trial of Revascor.
The study was conducted at one US centre on 19 children with HLHS and accepted for publication in an upcoming issue of The Journal of Thoracic and Cardiovascular Surgery Open (JTCVS Open).
In the trial, a single intramyocardial administration of Revascor at the time of staged surgery significantly increased LV systolic and diastolic volumes over a 12-month period in comparison to the control group.
These changes indicate the small left ventricle’s clinically significant enlargement, which may aid in a later surgical repair that restores the normal two ventricle circulation.
The enhancement in left ventricular functional results with Revascor may support more widespread use of surgical procedures to make a functioning left ventricle in children with HLHS.
This will result in reduced long-term morbidity and mortality when compared to alternative medicinal and/or surgical methods, the company said.
Additionally, Revascor was assessed in the DREAM-HF randomised sham-placebo controlled prospective trial of 565 adult patients with heart failure with low ejection fraction (HFrEF).
A single intramyocardial administration of Revascor into the left ventricle caused significant growth in LV ejection fraction at 12 months, suggesting strengthened overall LV systolic function.
Mesoblast is engaged in the development of product candidates for distinct indications using its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms.
