Mesoblast's Chief Executive, Silviu Itescu, expressed satisfaction with the FDA's decision: “We are very pleased to have now been granted both Orphan-Drug Designation and Rare Pediatric Disease Designation by FDA for Revascor® in the treatment of children with this often-fatal congenital heart condition."

The ODD was granted based on results from a randomized controlled trial demonstrating Revascor®'s potential to enhance the outcomes of life-saving surgery for children with HLHS. The trial, conducted in the United States and published in the December 2023 issue of The Journal of Thoracic and Cardiovascular Surgery Open (JTCVS Open), involved 19 children with HLHS.

The trial results showed that a single intramyocardial administration of Revascor® during staged surgery led to significantly larger increases in left ventricular (LV) end-systolic and end-diastolic volumes over 12 months compared to controls, as measured by 3D echocardiography (p=0.009 & p=0.020 respectively).

These findings suggest that Revascor® treatment may facilitate the growth of the small left ventricle, increasing the likelihood of a successful surgical correction known as full biventricular (BiV) conversion. Without full BiV conversion, there is an elevated risk of heart failure and death due to the strain on the right heart chamber.

Mesoblast intends to engage with the FDA to discuss the pathway for approval of Revascor® in treating children with HLHS, emphasizing the potential of this therapy to address an unmet medical need and improve outcomes for pediatric patients with congenital heart disease.