Unprecedented Move: US FDA Gives Mesoblast the Go-Ahead For Children’s Graft Versus Host Disease Therapy

Mesoblast Limited, a global leader in allogeneic cellular medicines for inflammatory diseases, just received a complete response from the US Food and Drug Administration (FDA) with regards to their resubmitted Biologics License Application (BLA) for remestemcel-L for the treatment of pediatric steroid-refractory acute graft versus host disease (SR-aGVHD). However, the FDA has requested more data to support their marketing approval.

To satisfy this requirement, Mesoblast will launch a targeted, controlled study with the highest-risk, critically ill adult patients. While the goal of the study is to support remestemcel-L’s candidacy, this also supports their commercial strategy which intends to progress from pediatric to adult SR-aGVHD indication; given that adults comprise 80% of SR-aGVHD incidence.

Mesoblast Chief Executive Silviu Itescu expressed his commitment to make remestemcel-L available to those suffering from a devastating disease, which has impacted more than 1300 patients to date. He added that the FDA’s inspection of their manufacturing process raised no safety issues and acknowledged the improvements to their potency assay. Through this, Itescu reassured those looking for relief that remestemcel-L is a product that can help them.

Mesoblast is on a mission to improve the lives of adult patients at highest mortality risk with SR-aGVHD. Where traditional therapies have failed to provide a response, Mesoblast has generated exciting preliminary data with remestemcel-L that shows a potential survival benefit. In alignment with their commercial strategy, the company is now working with renowned US centers of excellence to create a study protocol for adult SR-aGVHD. With the aim of obtaining FDA approval, Mesoblast is planning on a Type A meeting within the next 45 days to reaffirm their trial design.

Mesoblast received clear instructions from the FDA to resolve outstanding chemistry, manufacturing and controls (CMC) issues before initiating any additional clinical trial. Following the Pre-License Inspection (PLI) of the manufacturing facility, FDA found no objectionable conditions and did not issue any Form 483. Greatly encouraged by the results, FDA also noted that the new assay performed better than the original version used in the pediatric Phase 3 trial.

Mesoblast’s Phase 3 trial in 54 children with SR-aGvHD has been a success as it met the pre-specified primary endpoint prospectively agreed with the FDA. The company’s resubmission of January 2023 made a powerful impact as it included long-term follow-up data from the Phase 3 trial conducted by the Center for International Blood and Marrow Transplant Research (CIBMTR), showing 50% survival through more than 4 years of follow-up.

Furthermore, a post-hoc propensity matched study indicated 6-month survival of 67% for remestemcel-L treated patients compared to 10% for other unapproved therapies in highest-risk patients. These impressive results provide compelling evidence for the efficacy of remestemcel-L in the proposed study in high-risk adults with SR-aGVHD.

About Steroid-Refractory Acute Graft Versus Host Disease

Allogeneic bone marrow transplants (BMTs) are a life-saving treatment for blood cancers, with over 30,000 cases occurring worldwide every year, including 20% among pediatric patients. Unfortunately, up to half of these patients experience acute GVHD, a potentially life-threatening complication. Despite this, many patients are able to successfully undergo BMTs and enjoy a better quality of life as a result.

Systemic corticosteroids are the initial line of treatment for SR-aGVHD. Unfortunately, for many patients, this medication does not prove effective, leading to an alarming mortality rate of up to 90%, as well as substantial hospitalization costs. This makes SR-aGVHD a complex and serious condition to deal with.

About Mesoblast

Mesoblast is revolutionizing the medical landscape with the development of innovative allogeneic cellular medicines to combat severe and life-threatening inflammatory conditions. Utilizing its cutting-edge mesenchymal lineage cell therapy technology platform, the company has built an impressive portfolio of late-stage product candidates that have the power to reduce destructive inflammation by releasing anti-inflammatory factors that modulate the immune system – thus providing significant relief.

Mesoblast’s highly innovative approach to cell therapy has resulted in an unrivalled patent portfolio, securing the rights to these revolutionary therapies through to 2041 in major global markets. Not only does this bring forth the promise of accessible, off-the-shelf treatments, but Mesoblast’s state-of-the-art manufacturing process guarantees that these cell therapies can be delivered in an industrial-scale, cryopreserved form with precisely defined pharmaceutically-release criteria.

Mesoblast is incredibly passionate about pioneering therapeutic treatments that improve the quality of life of patients suffering from various inflammatory diseases, heart failure and chronic pain. The company has developed innovative allogeneic stromal cell technology platforms, Remestemcel-L and Rexlemestrocel-L, areas that have seen amazing success with two of their commercialized products in Japan and Europe. What’s even more encouraging is that Mesoblast is now focusing on further expanding its reach through Europe and China in Phases 3 of their projects.

Mesoblast, a global biotechnology enterprise listed on the Australian Securities Exchange (MSB) and the Nasdaq (MESO), has operations on three continents. Based in Australia, the company also has a presence in the United States and Singapore, enabling it to pursue innovative treatments for some of the world’s most challenging diseases.

Forward-Looking Statements

Mesoblast is making forward-looking statements about its preclinical and clinical studies, research and development programs, manufacturing capabilities, regulatory filings and approvals, commercialization of product candidates, and its strategic partnerships. By taking into consideration known and unknown risks, we aim to benefit from the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and any other federal securities laws that may be applicable.

These forward-looking statements involve potential patient safety, regulatory approvals, and market acceptance of stem-cell based therapies and the potential for Mesoblast’s product candidates, if approved, to be removed from the market due to any adverse events or deaths. Furthermore, Mesoblast plans to protect its intellectual property rights and to monitor its competitors and industry.

Given these uncertainties and risks, investments in our product candidates may yield material and adverse outcomes and our product candidates, if approved, may not result in expected benefits. Mesoblast promises to update or revise any future developments in a timely manner.

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