ISCT Hails Landmark US FDA Approval of RYONCIL as Major Milestone for MSC Field

VANCOUVER, BC, Dec. 20, 2024 /PRNewswire/ - The International Society for Cell & Gene Therapy (ISCT) celebrates the US Food and Drug Administration's (FDA) landmark decision to approve Mesoblast Limited's allogenic, bone marrow-derived mesenchymal stromal cell (MSC) product, RYONCIL® (remestemcel-L) for pediatric, steroid-refractory, acute Graft-versus-Host-Disease (GvHD). This decision is a watershed moment for the MSC field, marking the first approval of an MSC therapy in the US.

Around 50% of pediatric patients receiving an allogeneic bone marrow transplant develop acute GvHD, where transplanted cells recognize the host body as foreign and attack the patient's tissues and organs. Half of these patients do not respond to first-line steroid treatment. RYONCIL® will now provide a clinically effective option to meet the significant mortality and life-changing impacts of steroid-refractory acute GvHD.

Sowmya Viswanathan, PhD, ISCT MSC Committee Chair, spoke to the patient perspective, "The approval of RYONCIL addresses an urgent and unmet need for pediatric patients. Acute GvHD is a painful condition, and as organ failure spreads, quality of life deteriorates rapidly. This approval is a victory for patients and families who can now look towards a viable treatment option against a debilitating condition." 

Mesoblast Limited secured the approval on their third Biologics License Application (BLA) resubmission, working closely with the US FDA to address concerns after initial submissions were rejected in 2020 and 2023 respectively. Clinical efficacy was based on a single-arm, 54 patient, Phase III trial (NCT02336230) using a day 28 overall response, which was 70.4% compared to preset control group of 45%.

 "While MSC products first arrived to market over twelve years ago in other jurisdictions, this first approval in the United States is of historical importance," noted Jacques Galipeau, MD, ISCT Immediate Past President, "it rewards the work of researchers, clinicians, and innovators around the globe who have dedicated their careers to this field."

Speaking on this important approval, Miguel Forte, MD, PhD, ISCT President, stated "ISCT commends the resilient efforts that the team at Mesoblast has put into acquiring regulatory approval through hard-won proof of clinical efficacy.  This is a victory for the field, as enthusiasm and investment towards research and development of MSC therapies will be reignited. We may look back on this approval as a pivotal moment in the history of medicine, shaping the future of therapeutics."

About the International Society for Cell & Gene Therapy

Established in 1992, the International Society for Cell & Gene Therapy (ISCT) is a global society of clinicians, regulators, researchers, technologists, and industry partners with a shared vision to translate cell and gene therapy into safe and effective treatments to improve lives worldwide.

ISCT is the global leader focused on pre-clinical and translational aspects of developing cell and gene-based therapeutics, thereby advancing scientific research into innovative treatments for patients. ISCT offers a unique collaborative environment that addresses three critical areas of translation: Academia, Regulatory, and Commercialization. Through solid relationships with global regulatory agencies, academic institutions, and industry partners, ISCT drives the advancement of research into a standard of care.

Comprising over 4,000 cell and gene therapy experts across five geographic regions and with representation from over 60 countries, ISCT members are part of a global community of peers, thought leaders, and organizations invested in cell and gene therapy translation. For more information about the society, key initiatives, and upcoming meetings, please visit https://isctglobal.org, @ISCTglobal.

SOURCE International Society for Cell & Gene Therapy