FDA Deems Phase 3 Data Sufficient for Remestemcel-L BLA in Pediatric SR-aGVHD

Mesoblast Limited, a leading global developer of allogeneic cellular therapies for inflammatory diseases, has received a positive update from the U.S. Food and Drug Administration (FDA) regarding its Phase 3 study MSB-GVHD001. The FDA has indicated that the clinical data from this study is adequate to support the submission of a Biologics License Application (BLA) for remestemcel-L, a treatment for pediatric patients with steroid-refractory acute graft versus host disease (SR-aGVHD).

The company's CEO, Dr. Silviu Itescu, expressed gratitude for the FDA's collaborative approach and noted the clear guidance provided, which has bolstered the company's confidence in resubmitting the BLA for remestemcel-L. Mesoblast plans to resubmit the application in the coming quarter, with the aim of resolving all outstanding product characterization concerns.

Mesoblast is renowned for its development of allogeneic, or off-the-shelf, cellular medicines to treat severe and life-threatening inflammatory conditions. The company's proprietary technology platform has led to a robust pipeline of late-stage product candidates. These treatments work by releasing anti-inflammatory factors that counteract and modulate various aspects of the immune system, leading to a significant reduction in damaging inflammation.

The company also boasts a comprehensive global intellectual property portfolio, with protections in place until at least 2041 across all major markets. Mesoblast's manufacturing processes are designed to produce large-scale, cryopreserved, and readily available cellular medicines that meet defined pharmaceutical release criteria.

Mesoblast's product development is focused on distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell platforms. Remestemcel-L is being developed to treat inflammatory conditions in both children and adults, including SR-aGVHD and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is targeted towards advanced chronic heart failure and chronic low back pain. The company's products have been commercialized in Japan and Europe through licensing agreements, and Mesoblast has established partnerships in Europe and China for certain Phase 3 assets.