December 5, 2024 (Melbourne, Australia & New York, USA): Mesoblast Limited (ASX: MSB; Nasdaq: MESO), a global leader in allogeneic cellular medicines, has announced that the FDA has granted its second-generation stromal cell therapy, Revascor® (rexlemestrocel-L), the Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of hypoplastic left heart syndrome (HLHS) in children.
Key Highlights:
- FDA RMAT Designation:
- The RMAT designation was granted following the submission of results from a randomized controlled trial in children with HLHS, a life-threatening congenital heart condition.
- This designation provides a pathway for expedited development of therapies aimed at serious or life-threatening conditions where preliminary evidence shows potential to address unmet medical needs.
- Previous Designations:
- Earlier in 2024, Revascor® received Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) for the treatment of HLHS. These designations recognize the disease as serious and life-threatening, affecting children, including neonates and infants.
- Priority Review Voucher (PRV): If Revascor® is approved for HLHS, Mesoblast may be eligible to receive a PRV, which can expedite future approvals for other therapies.
- Trial Results:
- Results from the December 2023 study, published in The Journal of Thoracic and Cardiovascular Surgery Open, showed significant improvements in the left ventricular volumes of children treated with Revascor®.
- The trial involved 19 children with HLHS who received a single intramyocardial administration of Revascor® during staged surgery. The treatment led to significantly larger increases in left ventricular (LV) volumes, crucial for the success of subsequent surgical correction.
- Clinical Significance:
- The improvement in left ventricular volume is critical as it enables full biventricular conversion—a surgery that allows the heart to function normally. This reduces the strain on the right heart chamber, lowering the risk of heart failure, liver failure, and death.
CEO Remarks:
Mesoblast CEO Silviu Itescu expressed appreciation for the FDA’s support, stating that the RMAT and RPDD designations recognize the potential impact of Revascor® on the long-term outcomes for children with HLHS. The company plans to meet with the FDA to discuss the approval pathway for the therapy.
Regenerative Medicine Advanced Therapy (RMAT):
The RMAT designation is designed to expedite the development of regenerative therapies that may treat, modify, reverse, or cure serious conditions. It provides all the benefits of Breakthrough Therapy and Fast Track designations, such as rolling reviews and priority review of Biologics License Applications (BLA).
