After three years of waiting, Mesoblast has resubmitted its biologic license application for Ryoncil (remestemcel-L), a potential treatment for children suffering from steroid-refractory acute graft versus host disease (SR-aGVHD). If approved, Ryoncil could offer hope to those who have not responded to traditional treatments.
Hope is being renewed for this treatment, as it is given a second chance. This time, there is a chance to make it successful, so that everyone can benefit from its potential. Everyone involved is eager to see the outcome of this new opportunity and to see if it will make a difference in the lives of those affected by the condition.
The FDA has set an ambitious goal – to make Aug. 2, 2023 the day that allogeneic “off-the-shelf” cellular medicine and therapy is available in the U.S. for children under 12 with SR-aGVHD, a life-threatening disease. This groundbreaking step could revolutionize the treatment of this debilitating illness and save countless lives.
In October 2020, the FDA delivered a crushing blow to patients suffering from advanced pancreatic cancer, rejecting the highly anticipated drug that the Oncologic Drugs Advisory Committee had voted 9 to 1 in favor of. This decision highlighted the need for further research and development to find treatments for this life-threatening disease.
Every year, an estimated 300,000 patients undergo an allogeneic bone marrow transplant, and 20% of those are pediatric patients. Of those who receive the transplant, 50% will be at risk for acute GVHD, making it a serious consideration for those considering a bone marrow transplant.
The mortality rate of SR-aGVHD is a staggering 90%, making it one of the most serious and life-threatening conditions.
The FDA rejected Mesoblast’s application for their potentially life-changing drug due to lack of evidence that it could effectively treat steroid-refractory acute graft-versus-host disease (SR-aGVHD). To prove its efficacy, the FDA requested an additional randomized control trial in both adults and children.
Mesoblast has further strengthened its initial submission with the resubmission of consistently positive clinical outcomes for 309 children treated with Ryoncil. This resubmission further demonstrates the efficacy of the treatment and its potential to help children suffering from severe respiratory conditions.
A new study has just been released that compares the outcomes of 25 children who participated in a Phase III trial with those of 27 control children who were treated with various biologics. The study used validated MAP biomarker scores to measure the results, providing valuable insights into the effectiveness of different treatments.
In just 28 days, 67% of high-risk children showed positive responses to treatment and remained alive after 180 days – a remarkable improvement compared to the mere 10% in the same categories found in the Mount Sinai database.
The FDA has granted Ryoncil Fast Track and Priority Review designations in response to the new data submissions, providing a much-needed boost to the process for this promising treatment.
Ryoncil is a revolutionary cell therapy derived from the bone marrow of an unrelated donor. Its culture-expanded mesenchymal stromal cells have the potential to suppress the inflammatory processes of SR-aGVHD, offering a novel immunomodulatory treatment.
The aim of this therapy is to reduce inflammation by decreasing pro-inflammatory cytokines and increasing anti-inflammatory cytokines, as well as summoning naturally-occurring anti-inflammatory cells to the site. This innovative approach offers the potential to reduce inflammation and promote healing.