FDA Grants RMAT Designation To Mesoblast's Revascor for Pediatric Hypoplastic Left Heart Syndrome

Mesoblast Limited announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its Revascor (rexlemestrocel-L), a second-generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell therapy. This decision comes after the submission of results from a randomized controlled trial involving children with hypoplastic left heart syndrome (HLHS), a potentially life-threatening congenital heart condition.

Earlier in the year, Revascor received both Rare Pediatric Disease Designation (RPDD) and Orphan-Drug Designation (ODD) from the FDA for the treatment of HLHS in children. The RPDD underscores the severity of HLHS, which is a serious or life-threatening condition primarily affecting individuals from birth to 18 years old, including neonates, infants, children, and adolescents. It also confirms that HLHS is a rare disease or condition.

Upon FDA approval of a Biologics License Application (BLA) for Revascor for HLHS treatment, Mesoblast could be eligible for a Priority Review Voucher (PRV). This voucher can be used for any subsequent marketing application or sold/transferred to a third party, offering a significant advantage in the drug development and approval process.