Mesoblast has announced that the US Food and Drug Administration (FDA) has accepted a resubmitted biologics license application (BLA) for its pediatric steroid-refractory acute graft versus host disease (SR-aGvHD) therapy, remestemcel-L.
As per the 23 July press release, the latest resubmission addressed the remaining chemistry, manufacturing, and control (CMC)-related concerns raised by the FDA in March 2024. Moreover, the agency concluded that the available data from the Phase III study (NCT02336230) was “sufficient” to support the BLA in this paediatric population.
The agency will now take action by the Prescription Drug User Fee Act (PDUFA) date of January 7, 2025. If approved, remestemcel-L would become the first allogeneic stem cell therapy available in the US for the treatment of children with SR-aGvHD, the company shared in the announcement.
The BLA is based on data from an open label, single-arm Phase III study, which evaluated remestemcel-L in 55 pediatric patients who had failed to respond to prior steroid treatment for acute GvHD. Published results reported that at Day 28, treatment with remestemcel-L demonstrated a 70% improvement in overall response rate (ORR) compared to the 45% observed in the prespecified control. The statistically significant ORR was sustained through Day 100.
Mesoblast has traversed a tumultuous submission path for remestemcel-L. After resubmitting the BLA in January 2023 with additional data requested by the FDA, the agency issued a complete response letter (CRL) in August 2023 noting that more data was needed to support a marketing approval. The biotech subsequently announced plans to conduct a Phase III study that would include adults with the highest disease status and risk.
Following the CRL, the Melbourne, Australia-based biotech announced a partnership with the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) to develop and investigate remestemcel-L in patients aged 12 years and older with SR-aGvHD who have failed to respond to prior corticosteroid and second-line treatments.
Remestemcel-L is an intravenously administered stem cell therapy developed with the mesenchymal stem cells (MSC) from the bone marrow of an unrelated donor. The therapy is designed to decrease the production of pro-inflammatory cytokines that drive tissue damage associated with acute GvHD.
The therapy is approved for pediatric use in this indication and marketed under the brand name Temcell in Japan and Prochymal in Canada and New Zealand.
According to GlobalData’s consensus forecasts, remestemcel-L is expected to generate total sales of $379m in 2030. GlobalData is the parent company of Pharmaceutical Technology.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.
