On Monday, Mesoblast Ltd. MESO announced alignment with the U.S. Food and Drug Administration (FDA) on items required for filing a Biologics License Application (BLA) for Revascor (rexlemestrocel-L) for ischemic heart failure with reduced ejection fraction (HFrEF) and inflammation.
In follow-up to the successful Type B meeting in early 2024 under the existing Regenerative Medicine Advanced Therapy (RMAT) designation for Revascor in end-stage HFrEF patients with a left ventricular assist device (LVAD), where the FDA stated that the results of the presented studies could support accelerated approval, Mesoblast met with FDA on June 3, 2025 to align on key items for BLA filing.
The FDA provided formal minutes to the company following the most recent Type B meeting. The minutes confirm alignment with FDA on chemistry, manufacturing & controls (CMC) items, potency assays for commercial product release, and proposed design and primary endpoint for the confirmatory trial post-approval.
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Mesoblast intends to file for accelerated approval in patients with end-stage ischemic HFrEF implanted with an LVAD by the end of the year.
In other recent developments, Mesoblast received seven years of orphan-drug exclusive approval from the FDA for Ryoncil (remestemcel-L) for steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older in May.
This period of statutory exclusivity means that the FDA will not approve another mesenchymal stromal or stem cell (MSC) products for this indication during the 7 years from the approval of Ryoncil.
Separately, Mesoblast has biologic exclusivity preventing another sponsor from referencing the Ryoncil biologic license application (BLA) until December 2036, twelve years from its first approval which would prevent market entry by a biosimilar.
These statutory exclusivities, in addition to Mesoblast’s strong U.S. intellectual property position on MSC composition of matter, manufacturing, and indications, including SR-aGvHD, provide a commercial barrier to entry against competitors through 2044.
In early July, Mesoblast will meet with the FDA to discuss a pivotal trial of Ryoncil in adults with SR-aGvHD.
This trial will be conducted with the NIH-funded Bone Marrow Transplant Clinical Trials Network (BMT-CTN). The objective is to extend the product’s label from children to adults with SR-aGvHD.
Price Action: At the last check on Tuesday, MESO stock was up 8.82% at $11.85 during the premarket session.
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