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Highlights
- Mesoblast has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for its second-generation allogeneic stromal cell therapy Revascor® (rexlemestrocel-L) in treating children with Hypoplastic Left Heart Syndrome (HLHS).
- This designation follows the results of a successful randomized controlled trial, which showed significant improvements in left ventricular volumes in children with HLHS.
- Revascor® also holds Rare Pediatric Disease Designation (RPDD) and Orphan-Drug Designation (ODD) from the FDA, marking it as a promising treatment for this serious, life-threatening congenital heart condition.
Mesoblast Limited (Nasdaq:MESO; ASX: MSB), a global leader in allogeneic cellular medicines for inflammatory diseases, today announced that the United States Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its second-generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell therapy, Revascor® (rexlemestrocel-L). This designation follows the submission of promising results from a randomized controlled trial in children with Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart condition.
The RMAT designation is a significant milestone for Mesoblast and highlights the potential of Revascor® to address the unmet medical needs of children suffering from HLHS. Under the RMAT designation, Mesoblast plans to engage with the FDA to discuss an accelerated pathway for regulatory approval.
FDA Designations Support Revascor®’s Promise in Treating HLHS
Earlier this year, the FDA granted Revascor® both Rare Pediatric Disease Designation (RPDD) and Orphan-Drug Designation (ODD) for the treatment of HLHS. The RPDD acknowledges the seriousness of HLHS, which primarily affects children from birth to 18 years and often results in life-threatening complications.
The RMAT designation for Revascor® builds on this momentum and provides Mesoblast with additional benefits, including eligibility for priority review and rolling review upon filing a Biologics License Application (BLA). Furthermore, if the BLA for Revascor® is approved by the FDA for the treatment of HLHS, Mesoblast may be eligible to receive a Priority Review Voucher (PRV), which can be redeemed for any subsequent marketing application or sold to a third party.
Promising Clinical Results for Revascor® in HLHS Trial
In the randomized, blinded, placebo-controlled trial conducted in the United States, Revascor® demonstrated positive results in 19 children with HLHS. A single intramyocardial administration of Revascor® at the time of staged surgery resulted in significantly larger increases in left ventricular (LV) end-systolic and end-diastolic volumes over 12 months when compared to controls, as measured by 3D echocardiography. These results were statistically significant with p-values of 0.009 and 0.020, respectively.
These improvements are highly promising, as HLHS is characterized by underdeveloped left heart structures, leading to limited blood flow and severe long-term cardiovascular complications. By enhancing heart function, Revascor® may significantly improve the long-term health outcomes of children with this condition.
Mesoblast’s CEO Comment
Silviu Itescu, CEO of Mesoblast, commented: “We appreciate the FDA’s recognition of Revascor®’s potential in treating HLHS with the RMAT and RPDD designations. These designations validate the promising data we have seen in the trial and highlight the significant unmet need for effective therapies for these critically ill children. We look forward to collaborating with the FDA to explore an accelerated regulatory path for Revascor® and aim to bring this life-saving therapy to children with HLHS as soon as possible.”
Looking Ahead
With these key FDA designations, Mesoblast is positioned to further advance the development of Revascor® for HLHS. The company will now focus on continuing its discussions with the FDA to map out the next steps toward obtaining approval for this groundbreaking therapy.
