Mesoblast (ASX:MSB) announced that the U.S. Food and Drug Administration (FDA) has granted advanced regenerative medicine therapy designation to its drug Revascor (rexlemestrocel-L) for children with hypoplastic left heart syndrome (HLHS).
This builds on the FDA’s previous designations of rare pediatric disease (RPD) and orphan drug (OD) status for the therapy.
Mesoblast develops allogeneic (ready-to-use) cellular medicines for the treatment of serious and life-threatening inflammatory conditions. These therapies target diseases by modulating immune system responses and reducing harmful inflammation.
Hypoplastic left heart syndrome
HLHS is a serious heart defect in which a baby is born with an underdeveloped left side of the heart, including the left ventricle, aorta, and mitral valve. This prevents the heart from effectively pumping oxygenated blood around the body. Without immediate surgery, the defect is usually fatal.
The syndrome affects approximately 2-3 newborns per 10,000 live births. It represents 2-9% of all congenital heart defects and is responsible for approximately 25-40% of neonatal cardiac deaths.
Revascor demonstrated promising results in a randomized, controlled trial involving 19 children with HLHS. Its effects include:
- promote the formation of new blood vessels in the underdeveloped left ventricle (“neovascularization”);
- reducing inflammation and preventing the immune system from attacking healthy tissue, creating an environment conducive to tissue growth (“immunomodulation”).
State of Advanced Regenerative Medicine Therapy (RMAT).
The FDA’s RMAT designation accelerates the development of therapies that address life-threatening conditions.
Benefits include continuous review, priority review eligibility, and expedited regulatory pathways for a biologics license application (BLA). Mesoblast plans to work with the FDA on an approval strategy.
CEO Silviu Itescu commented: “The FDA support recognizes Revascor’s potential impact on long-term outcomes for critically ill children. We look forward to advancing this therapy to address their unmet needs.”
Mesoblast shares surprisingly fell following the announcement. They are currently 7.1% lower at $1.64.