FDA Accepts Mesoblast's New BLA for Pediatric GvHD Cell Therapy

Mesoblast has achieved a significant milestone with the US Food and Drug Administration (FDA) accepting its resubmitted biologics license application (BLA) for remestemcel-L, a stem cell therapy designed for pediatric patients with steroid-refractory acute graft versus host disease (SR-aGvHD). This development follows the company's efforts to address the FDA's previously raised concerns regarding chemistry, manufacturing, and control (CMC) aspects.

The latest resubmission, as revealed on July 23, responded effectively to the FDA's concerns highlighted in March 2024. The agency determined that the data from the Phase III study (NCT02336230) were adequate to support the BLA for this pediatric population. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of January 7, 2025. If the therapy gains approval, it could become the first allogeneic stem cell therapy in the US for treating SR-aGvHD in children.

The data supporting the BLA come from a single-arm, open-label Phase III study involving 55 pediatric patients who had not responded to prior steroid treatments for acute GvHD. Results showed that by Day 28, remestemcel-L achieved a 70% overall response rate (ORR), compared to a 45% ORR in the prespecified control group. This statistically significant ORR remained consistent through Day 100.

However, Mesoblast's journey to this point has been challenging. After an initial BLA submission in January 2023, the FDA issued a complete response letter (CRL) in August 2023, requesting additional data for marketing approval. In response, Mesoblast announced plans to conduct a Phase III study encompassing adult patients with severe disease status and risk.

Following the CRL, the Melbourne-based biotech company partnered with the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) to further develop and evaluate remestemcel-L in patients aged 12 and older who have not responded to prior corticosteroid and second-line treatments.

Remestemcel-L is administered intravenously and utilizes mesenchymal stem cells (MSC) derived from the bone marrow of an unrelated donor. The therapy aims to reduce the production of pro-inflammatory cytokines that cause tissue damage in acute GvHD. It is already approved for pediatric use in certain regions, marketed as Temcell in Japan and Prochymal in Canada and New Zealand.

Remestemcel-L represents a promising therapeutic development in the field of stem cell therapy, particularly for pediatric patients facing the challenging condition of SR-aGvHD. The successful acceptance of the BLA by the FDA marks a crucial step towards potentially offering a new, effective treatment option for children in need.

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