Mesoblast Secures FDA RMAT Designation for Congenital Heart Disease Treatment
Thursday, December 05, 2024
Mesoblast has announced that the US Food and Drug Administration (FDA) has granted its stromal cell therapy, Revascor® (rexlemestrocel-L), the Regenerative Medicine Advanced Therapy (RMAT) designation.
Revascor is an allogeneic preparation of mesenchymal precursor cells with multiple therapeutic mechanisms, including neovascularisation, immunomodulation, and reduction of inflammation and fibrosis. In a previous study involving adults with heart failure and low ejection fraction (HFrEF), Revascor demonstrated improvements in left ventricular function and reduced adverse cardiovascular events, particularly in high-risk patients.
HLHS is a severe congenital defect in which the left side of the heart is underdeveloped, impairing its ability to pump oxygenated blood effectively. Without immediate surgical intervention, survival prospects are extremely poor, with HLHS contributing to 25–40% of all neonatal cardiac mortality. Surgical repair aimed at establishing a two-ventricle circulation is the ideal approach, but this is often limited by insufficient growth of the left ventricle.
This recognition follows promising results from a clinical trial in children with hypoplastic left heart syndrome (HLHS), a severe congenital heart condition.
Earlier this year, Revascor also received Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) from the FDA for treating HLHS. These designations highlight the therapy's potential to address significant medical needs in rare and life-threatening paediatric conditions.
Upon FDA approval of a Biologics License Application (BLA) for Revascor in HLHS, Mesoblast may qualify for a Priority Review Voucher (PRV), which can expedite future regulatory reviews or be transferred to another entity.
RMAT designation is aimed at accelerating the development of regenerative medicine therapies that target serious or life-threatening conditions, where preliminary evidence suggests a potential to meet unmet medical needs.
It offers benefits such as rolling review and priority review eligibility upon BLA submission.
The recent trial conducted in the US involved 19 children with HLHS and showed positive results. A single intramyocardial administration of Revascor during surgery significantly increased left ventricular (LV) end-systolic and end-diastolic volumes over 12 months, as measured by 3D echocardiography.
These improvements were associated with improved growth of the underdeveloped left ventricle, facilitating the potential for a successful biventricular (BiV) surgical correction. Achieving BiV correction reduces the strain on the right heart chamber, lowering risks of heart failure, liver failure, and mortality.
These findings underscore Revascor’s potential as a transformative therapy for both paediatric and adult heart conditions.
Source: mesoblast.com
