Mesoblast’s Resubmission Brings First-of-its-Kind Therapy One Step Closer to Approval

The US FDA has accepted Mesoblast’s Biologics License Application (BLA) resubmission for remestemcel-L in the treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD), a life-threatening complication of an allogeneic bone marrow transplant for blood cancer. The regulatory review is expected to be completed on August 2, 2023. 

The acceptance is a relief for New York-based Mesoblast, after the company was hit with a complete response letter for remestemcel-L in October 2020. The FDA recommended then that Mesoblast conduct an extra study to provide further evidence of the effectiveness of the cell therapy. 

“Over the last two years we have worked tirelessly to address the issues previously raised by FDA. We look forward to working closely with the Agency over the review period with the aim to make remestemcel-L available as a therapy for children suffering from SR-aGVHD,” said Mesoblast CEO Silviu Itescu.

If approved, Remestemcel-L could become the first off-the-shelf cell-based medicine in the US, and the first therapy for children under 12 years of age with SR-aGVHD. The therapy holds designations for Fast Track and Priority Review, which could speed up the regulatory process. 

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Remestemce-L Associated with Positive Responses and Survival

Remestemcel-L contains culture-expanded mesenchymal stromal cells derived from the bone marrow of an unrelated donor. Administered intravenously, the therapy may help counteract the inflammation in SR-aGVHD by down-regulating the production of pro-inflammatory cytokines, increasing the production of anti-inflammatory cytokines, and enabling the recruitment of anti-inflammatory cells.

Mesoblast’s original filing included data from three trials showing consistent treatment responses and survival with remestemcel-L treatment. 

The resubmission adds more clinical and biomarker data, including from a study of children with high-risk disease. The study showed that 67% of high-risk children treated with remestemcel-L responded positively to treatment and were alive after 180 days compared to 10% of children treated with various biologics such as ruxolitinib. 

Additionally, a four-year survival study performed by the Center for International Blood and Marrow Transplant Research (CIBMTR) showed that 63% of children treated with remestemcel-L survived at one year while 51% survived at two years. The results compared to an expected two-year survival rate of 25-38% using the best available therapy. 

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Joy Lin

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