Key Highlights:
- FDA RMAT designation granted to Revascor® (rexlemestrocel-L) for treatment of children with Hypoplastic Left Heart Syndrome (HLHS).
- Rare Pediatric Disease Designation (RPDD) and Orphan-Drug Designation (ODD) were granted earlier this year.
- Revascor® shows promising results in improving left ventricular growth, which is critical for successful heart surgery in children with HLHS.
- The therapy’s clinical trial results published in The Journal of Thoracic and Cardiovascular Surgery Open show significant improvement in key heart measurements.
Melbourne, Australia; December 5, 2024 – New York, USA; December 4, 2024 – Mesoblast Limited (ASX:MSB; Nasdaq:MESO), a global leader in allogeneic cellular medicines for inflammatory diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Revascor® (rexlemestrocel-L) for the treatment of children with Hypoplastic Left Heart Syndrome (HLHS). This recognition comes following positive results from a randomized controlled trial in children with this severe and potentially life-threatening congenital heart condition.
Silviu Itescu, CEO of Mesoblast, said: “We are grateful for the FDA’s recognition of Revascor® as a potential breakthrough treatment for children suffering from HLHS. The RMAT designation reflects the promising clinical data and the urgent need for effective therapies for these children. We look forward to engaging with the FDA on a potential approval pathway for Revascor®.”
Revascor®’s Clinical Breakthrough
Earlier in 2024, Revascor® received both Rare Pediatric Disease Designation (RPDD) and Orphan-Drug Designation (ODD) for its potential to address HLHS. These designations underscore the seriousness of the disease, which primarily affects neonates, infants, children, and adolescents.
The RMAT designation accelerates the development of treatments for serious conditions by offering the benefits of both Breakthrough Therapy and Fast Track status, including rolling review and priority review eligibility for a Biologics License Application (BLA).
The randomized controlled trial in 19 children with HLHS demonstrated that a single intramyocardial injection of Revascor® at the time of staged surgery led to significant improvements in left ventricular (LV) growth over 12 months, as measured by 3D echocardiography. These improvements were critical for enabling full biventricular (BiV) conversion, a life-saving surgical procedure that restores normal heart function. Without this growth, children are at risk of heart failure, liver failure, and death.
