Mesoblast Limited, a leader in cellular medicine for inflammatory conditions, has revealed that the US FDA has awarded their allogeneic stromal cell therapy, Revascor (rexlemestrocel-L), the Regenerative Medicine Advanced Therapy (RMAT) designation. This follows promising outcomes from a trial centered on children with Hypoplastic Left Heart Syndrome (HLHS), a serious congenital cardiac defect.
“We express our gratitude towards the FDA for acknowledging the promise of Revascor with both RMAT and Rare Pediatric Disease designations,” asserted Silviu Itescu, CEO of Mesoblast. “This recognition underscores the potential of our therapy to dramatically affect the long-term health of critically ill young patients.”
Earlier this year, Revascor also attained Rare Pediatric Disease Designation (RPDD) and Orphan-Drug Designation (ODD) for HLHS treatment. RPDD underscores the life-threatening nature of conditions affecting individuals from birth to 18 years and recognizes rare diseases or conditions. Upon FDA approval for its Biologic License Application (BLA), Mesoblast might obtain a Priority Review Voucher (PRV), which could either be utilized for marketing or sold to another entity.
RMAT designations serve to fast-track the development of therapies targeting severe diseases with an unmet medical need, similar to the benefits provided under Breakthrough and Fast Track statuses.
The outcomes from a prospective trial involving Revascor against a placebo, involving US children with HLHS, will appear in a December 2023 issue of The Journal of Thoracic and Cardiovascular Surgery Open (JTCVS Open). The trial involved 19 children, revealing that a single administration of Revascor led to substantial increases in left ventricular volumes over 12 months compared to controls.
These enhancements are crucial for the growth of the underdeveloped left ventricle, enabling successful full biventricular conversion, an essential surgical outcome that supports healthier circulatory functionality. Without this conversion, the right heart bears excessive load, leading to increased heart failure risks.
HLHS is a severe heart defect where the heart’s left side remains underdeveloped, impairing efficient blood pumping. Without prompt postnatal surgery, the prognosis is bleak, with HLHS contributing significantly to neonatal cardiac fatalities. A two-ventricle anatomical repair remains the ideal remedy but is often hindered by the limited developmental growth of the left ventricle.
Revascor consists of immunoselected and cultured mesenchymal precursor cells, which have demonstrated various therapeutic actions. In adults with heart failure, trials revealed Revascor’s potential to enhance LV systolic function, notably benefiting those with heart failure linked to ischemia and inflammation.