Mesoblast (MESO) is nearing a critical milestone with its biologics license application (BLA) for remestemcel-L (Ryoncil), targeting steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients. The FDA has set a Prescription Drug User Fee Act (PDUFA) decision date of January 7, 2025, for the resubmitted application.
If approved, remestemcel-L would become the first available treatment for children under 12 suffering from SR-aGVHD. This designation addresses a significant unmet medical need, as current treatment options are limited for this vulnerable population. The drug has also been granted Rare Pediatric Disease designation, further highlighting its importance in treating this rare condition.
Potential Impact of Approval
While the SR-aGVHD market is relatively small, approval of remestemcel-L could significantly enhance Mesoblast's valuation and establish the drug as a leading treatment option in this critical niche. Industry analysts maintain a consensus Buy rating for Mesoblast, with an average price target of $11, suggesting a 56% upside potential from current levels.
Mesoblast is a Melbourne-based biotechnology company specializing in regenerative medicine and cell-based therapies. In addition to remestemcel-L, the company is also developing rexlemestrocel-L (Revascor) for chronic heart failure and chronic low back pain. The FDA has granted Orphan Drug Designation to rexlemestrocel-L for treating pediatric congenital heart diseases.
Financial Position
In the second quarter, three hedge funds held shares in Mesoblast, with total stakes worth $1.18 million. Marshall Wace LLP is the largest shareholder, holding $562,343 worth of stock as of June 30.
