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.
