- Mesoblast (ASX:MSB) is granted ODD and RPDD from the US FDA for its allogeneic cell therapy, Revascor
- These designations stem from positive results in a randomised controlled trial involving children with HLHS, a life-threatening congenital heart condition.
- Revascor is an allogeneic preparation with multiple mechanisms of action
- The company is actively engaging with the FDA to advance the approval process and address the urgent need for effective HLHS treatments
- MSB last traded at 28 cents
Mesoblast (ASX:MSB) has been granted orphan-drug designation (ODD) and rare pediatric disease designation (RPDD) from the United States Food and Drug Administration (FDA) for its allogeneic cell therapy, Revascor.
These designations stem from positive results in a randomised controlled trial involving children with hypoplastic left heart syndrome (HLHS), a life-threatening congenital heart condition.
Revascor is an allogeneic preparation with multiple mechanisms of action.
“We are very pleased to have now been granted both Orphan-Drug Designation and Rare Pediatric Disease Designation by FDA for REVASCOR in the treatment of children with this often-fatal congenital heart condition,” Mesoblast CEO Silviu Itescu said.
“The designations were granted on the back of the results from children in a randomised controlled trial indicating that REVASCOR may increase the ability to successfully accomplish life-saving surgery.
“We plan to meet with FDA to discuss the pathway for approval in this indication.”
The FDA’s ODD program offers development incentives to Mesoblast, including market exclusivity, exemption from application fees, tax credits, and other potential support throughout the drug development process.
The company is actively engaging with the FDA to advance the approval process and address the urgent need for effective HLHS treatments.
MSB last traded at 28 cents.