ISCT Celebrates Historic FDA Approval of RYONCIL for Pediatric GvHD Treatment in the U.S.

A Major Advancements in MSC Therapies: ISCT Celebrates FDA Approval of RYONCIL

The International Society for Cell & Gene Therapy (ISCT) recently applauded the U.S. Food and Drug Administration's (FDA) groundbreaking decision to approve RYONCIL® (remestemcel-L), an allogenic, bone marrow-derived mesenchymal stromal cell (MSC) therapy from Mesoblast Limited, specifically for pediatric patients with steroid-refractory acute Graft-versus-Host Disease (GvHD). This milestone signifies the first FDA approval of an MSC therapy in the United States, heralding a new era for therapies targeting this difficult-to-treat condition.

Acute GvHD primarily affects children who undergo allogeneic bone marrow transplants. Approximately 50% of these pediatric patients develop this condition as the transplanted cells attack their new host's body, recognizing it as foreign. For many, the initial steroid treatments prove ineffective. The approval of RYONCIL® offers a vital alternative, potentially saving lives and improving the quality of life for those affected.

Dr. Sowmya Viswanathan, Chair of the ISCT MSC Committee, emphasized the significance of this approval, stating, “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.”

Background on RYONCIL

The pathway to this monumental approval was challenging for Mesoblast Limited, which submitted three Biologics License Applications (BLA) to the FDA before securing the green light. Their successful approval comes after rigorous clinical trials, the most notable being a Phase III trial involving 54 patients, which demonstrated a 70.4% overall response rate by day 28. This was notably higher than the prespecified control group rate of 45% for the same time frame.

Dr. Jacques Galipeau, Immediate Past President of ISCT, noted the historical significance of this achievement: “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. It rewards the work of researchers, clinicians, and innovators worldwide who have dedicated their careers to this field.”

Miguel Forte, ISCT President, highlighted this approval as a pivotal moment in medicine. He remarked, “ISCT commends the resilient efforts that the team at Mesoblast has put into acquiring regulatory approval through extensive 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.”

The Future of MSC Therapies

This landmark decision is expected to inject new energy into research and development in the MSC therapy sector. It reflects the dedication and collaborative efforts of industry professionals committed to translating scientific advancements into tangible treatments that improve patient outcomes. As a global society established in 1992, ISCT remains at the forefront of developing cell and gene-based therapies, partnering with various stakeholders to drive research and innovation.

With over 4,000 members from 60 countries, ISCT fosters collaborative environments where academia, regulatory bodies, and industry professionals come together to establish cell and gene therapies as part of standard care. The promising advancements in MSC therapies, underscored by the approval of RYONCIL, exemplify ISCT's mission to better lives through safe and effective treatments.

In conclusion, the FDA's approval of RYONCIL is not only a moment of triumph but also a beacon of hope for countless families affected by pediatric acute GvHD. It sets a precedent for future innovative therapies in this critical area of medicine, underscoring the potential of MSC technologies.

For more information about ISCT and its commitment to advancing cell and gene-based therapeutic solutions, visit ISCT Global.