Mesoblast Limited (traded as MESO and MSB.AX) has announced that the U.S. Food and Drug Administration (FDA) has awarded its second-generation, industrially produced stromal cell therapy, Revascor (rexlemestrocel-L), with the Regenerative Medicine Advanced Therapy (RMAT) designation. This accolade comes on the heels of presenting results from a randomized controlled trial addressing its efficacy in children suffering from hypoplastic left heart syndrome (HLHS), a severe congenital heart defect.
Earlier this year, the FDA also recognized REVASCOR with both the Rare Pediatric Disease Designation (RPDD) and Orphan-Drug Designation (ODD) targeting pediatric patients afflicted with HLHS. The RPDD underscores the severe or life-threatening nature of this condition, which predominantly impacts individuals aged newborns to 18 years, spanning age groups often referred to as neonates, infants, children, and adolescents. Furthermore, it classifies HLHS as a rare disease or condition.
With FDA approval of a Biologics License Application (BLA) for REVASCOR’s treatment of HLHS, Mesoblast stands to potentially qualify for a Priority Review Voucher (PRV). Such a voucher can be utilized for expedited marketing application reviews or sold and transferred to another party.
