Mesoblast Resubmits BLA for Ryoncil to FDA for Pediatric SR-aGVHD Treatment
MSB (MSB) Share Update July 2024 Monday 8th
Mesoblast Resubmits Biologic License Application for Ryoncil with FDA
Mesoblast Limited (ASX:MSB; Nasdaq:MESO), a global leader in allogeneic cellular medicines, has announced the resubmission of its Biologic License Application (BLA) to the U.S. Food and Drug Administration (FDA) for Ryoncil® (remestemcel-L) to treat children with steroid-refractory acute graft-versus-host disease (SR-aGVHD).
Instant Summary:
- Mesoblast resubmits BLA for Ryoncil to FDA.
- Ryoncil aims to treat children with SR-aGVHD.
- FDA previously indicated clinical data supports BLA submission.
- Resubmission addresses remaining Chemistry, Manufacturing, and Control (CMC) items.
- Ryoncil granted Fast Track and Priority Review designations by FDA.
- Review period expected between two and six months.
FDA Resubmission Details
Mesoblast Limited has resubmitted its Biologic License Application (BLA) to the FDA for Ryoncil® (remestemcel-L), targeting the treatment of children with steroid-refractory acute graft-versus-host disease (SR-aGVHD). This resubmission follows the FDA's indication in March that the available clinical data from the Phase 3 study MSB-GVHD001 appears sufficient to support the BLA submission. The resubmission addresses remaining Chemistry, Manufacturing, and Control (CMC) items.
Dr. Silviu Itescu, CEO of Mesoblast, expressed gratitude towards the FDA for their ongoing guidance, emphasizing the urgent need for a therapy that improves survival outcomes in children with SR-aGVHD.
FDA Designations and Review Timeline
The FDA has granted remestemcel-L Fast Track designation, facilitating the development and expedited review of therapies for serious conditions with unmet medical needs. Additionally, remestemcel-L has received Priority Review designation, which is given to drugs that provide significant improvements in safety or effectiveness over existing treatments.
The BLA resubmission, upon acceptance, is expected to undergo a review period of between two and six months from receipt.
About Ryoncil® (remestemcel-L)
Ryoncil® (remestemcel-L) is an investigational therapy comprising culture-expanded mesenchymal stromal cells derived from the bone marrow of an unrelated donor. It is administered to patients via a series of intravenous infusions. Ryoncil has immunomodulatory properties that counteract inflammatory processes in SR-aGVHD by inhibiting the activation and proliferation of effector T cells, down-regulating pro-inflammatory cytokines, and enabling the recruitment of anti-inflammatory cells to affected tissues.
Phase 3 Trial Results
The Phase 3 Study GVHD001/002 involved 54 children across 20 centers in the US, where Ryoncil was used as the first line of treatment for children who failed to respond to steroids for acute GVHD. The trial met its primary endpoint, with a 70.4% overall response at Day 28 compared to 45% in the control group. This response was highly predictive of improved survival through Day 100.
Compared to a matched control group from the Mount Sinai Acute GVHD International Consortium (MAGIC) database, treatment with Ryoncil achieved higher Day 28 overall response (70% vs 43%) and higher Day 100 survival (74% vs 57%). A propensity-matched study further demonstrated significant survival benefits for high-risk children treated with Ryoncil.
About Steroid-Refractory Acute Graft Versus Host Disease
Acute GVHD occurs in approximately 50% of patients receiving an allogeneic bone marrow transplant (BMT), with over 30,000 patients undergoing BMT annually worldwide. SR-aGVHD is associated with high mortality rates and significant hospital stay costs. Currently, there are no FDA-approved treatments for children under 12 with SR-aGVHD, highlighting the urgent need for effective therapies.
About Mesoblast
Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory conditions. The company has a broad portfolio of late-stage product candidates and a strong global intellectual property portfolio. Mesoblast's proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf cellular medicines, planned to be readily available worldwide.
Mesoblast is developing remestemcel-L for inflammatory diseases in children and adults, including SR-aGVHD and biologic-resistant inflammatory bowel disease. The company also has commercial partnerships in Europe and China for certain Phase 3 assets and has commercialized two products in Japan and Europe through its licensees.
The resubmission of the BLA for Ryoncil is a significant step for Mesoblast, potentially leading to the approval of a much-needed therapy for children with SR-aGVHD. If approved, Ryoncil could improve survival outcomes and address a critical unmet medical need. This development is likely to positively impact Mesoblast's stock, as it demonstrates progress towards regulatory approval and potential market entry.
Investor Reaction:
Analysts and investors have shown optimism regarding the resubmission, noting the positive Phase 3 trial results and the FDA's supportive stance. The Fast Track and Priority Review designations further bolster confidence in the potential approval of Ryoncil.
Conclusion:
Investors should closely monitor the FDA's review process and potential approval of Ryoncil. The approval could significantly enhance Mesoblast's market position and provide a breakthrough therapy for children with SR-aGVHD. Stay informed on Mesoblast's progress and consider the potential long-term benefits of this development.