FDA grants Mesoblast orphan-drug designation for Revascor

Mesoblast Limited “announced that the United States Food and Drug Administration has granted its allogeneic cell therapy Revascor, or rexlemestrocel-L, an Orphan-Drug Designation following submission of results from the randomized controlled trial in children with hypoplastic left heart syndrome, a potentially life threatening congenital heart condition. This follows the Rare Pediatric Disease Designation granted by FDA last month. Results from a blinded, randomized, placebo-controlled prospective trial of REVASCOR conducted in the United States in children with HLHS were published in the December 2023 issue of the peer reviewed The Journal of Thoracic and Cardiovascular Surgery Open. In the HLHS trial conducted in 19 children, a single intramyocardial administration of REVASCOR at the time of staged surgery resulted in the desired outcome of significantly larger increases in left ventricular end-systolic and end-diastolic volumes over 12 months compared with controls as measured by 3D echocardiography. These changes are indicative of clinically important growth of the small left ventricle, facilitating the ability to have a successful surgical correction, known as full biventricular, BiV, conversion, which allows for a normal two ventricle circulation with the surgically repaired left ventricle taking over circulatory support to the body. Without full BiV conversion the right heart chamber is under excessive strain with increased risk of heart failure and death.”