Image source: Shutterstock

Highlights

  • FDA confirms alignment on CMC, potency assays, and confirmatory trial design.
  • Filing targeted by year-end for accelerated approval in end-stage ischemic HFrEF patients with LVAD.
  • Revascor holds RMAT designation for heart failure with inflammation 

Mesoblast (Nasdaq: MESO; ASX: MSB) announced that it has reached alignment with the U.S. Food and Drug Administration (FDA) on key components required to file a Biologics License Application (BLA) for its cell therapy candidate Revascor® (rexlemestrocel-L) for the treatment of ischemic heart failure with reduced ejection fraction (HFrEF) and inflammation. The agreement follows a Type B meeting with the FDA held on June 3, 2025. 

According to the company, the FDA has confirmed the agreement on the Chemistry, Manufacturing and Controls (CMC) package, potency assays for commercial release, and the design and primary endpoint of the confirmatory trial intended to be conducted post-approval. Mesoblast plans to file for accelerated approval by the end of 2025 for Revascor’s use in patients with end-stage ischemic HFrEF who are implanted with a left ventricular assist device (LVAD). 

The ongoing regulatory engagement follows a prior Type B meeting in early 2024 under the Regenerative Medicine Advanced Therapy (RMAT) designation. At that time, the FDA had indicated that data submitted by the company could support accelerated approval for this patient group. 

Mesoblast is a biotechnology company focused on developing off-the-shelf allogeneic cellular therapies targeting severe inflammatory diseases. The company’s technology platform centers on mesenchymal lineage cells that release anti-inflammatory factors in response to immune activation. In addition to Revascor, Mesoblast’s product portfolio includes RYONCIL® (remestemcel-L), which is FDA-approved for treating steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients. 

Mesoblast is also advancing remestemcel-L and rexlemestrocel-L for additional inflammatory and degenerative conditions, including biologic-resistant inflammatory bowel disease and chronic low back pain. The company maintains manufacturing infrastructure capable of producing cryopreserved, pharmaceutical-grade cell therapies at scale and has established commercial partnerships in markets including Japan, Europe, and China.