Interstuming stem cells (MSCs) treat grafts against host disease! Anti-inflammatory cell therapy Ryoncil was approved by a U.S. FDA panel of experts!

August 14, 2020 // -- Mesoblast Ltd, a Melbourne, Australia-based biotechnology company, is a global leader in isotrophysic (universal) cell therapies dedicated to the development of cell-based regenerative therapy products for the treatment of inflammatory diseases.
recently, the company announced that the U.S. Food and Drug Administration's (FDA) Advisory Committee on Oncology And Drugs (ODAC) voted overwhelmingly (9 in favor, 1 against) to support the effectiveness of the allogeneic cell therapy Ryoncil (remestemcel-L) for the treatment of childhood steroid incurable acute graft anti-host disease (SR-aGVHD).
, there is currently no FDA-approved treatment for children under 12 years of age with SR-aGVHD.
ODAC is an independent panel of experts responsible for assessing the validity and safety of data and providing appropriate advice to the FDA.
the FDA will consider the panel's opinion, the final decision on product approval is entirely up to the FDA and the panel's recommendations are non-binding.
, Ryoncil is currently under FDA priority review with a target date of September 30, 2020 for the Prescription Drug User Charge Act (PDUFA).
if approved, Mesoblast plans to bring Ryoncil to market immediately.
previously, the FDA had granted Ryancil fast-track eligibility (FTD) for treatment of SR-aGVHD. Dr Fred Grossman, chief medical officer of
Mesoblast, said: "Steroid incurable acute graft anti-host disease (SR-aGVHD) is an area of extreme need, especially in vulnerable children under 12 years of age, who do not have approved treatments.
we are very encouraged by today's results and are committed to working with the FDA to complete the review of Ryoncil's treatment for this life-threatening complication associated with allogeneic bone marrow transplantation.
"remestemcel-L is an allogeneic interstate charge stem cell (MSC) derived from MSC isolated in the bone marrow of an unrelevant donor and prepared for culture amplification.
remestemcel-L is administered intravenously and has an immunomodulation effect, fighting inflammatory processes associated with a variety of diseases by reducing the production of inflammatory cytokines, increasing the production of anti-inflammatory cytokines, and recruiting naturally occurring anti-inflammatory cells into related tissues.
acute graft anti-host disease (aGVHD) is a potentially life-threatening disease that occurs in about 50% of patients receiving allogeneic bone marrow transplantation (BMT).
, more than 30,000 patients worldwide receive the allogeneic BMT each year, mainly during the treatment of blood cancer, 20-25% of which are in children.
12-month mortality rate is as high as 90% in patients with the most severe type of aGVHD (C/D or III/IV).
, there is currently no FDA-approved treatment for children under 12 years of age with SR-aGVHD.
clinical trials were included in Ryoncil's Biological Products Licensing Application (BLA).
Ryoncil has been used in three clinical studies to treat 309 children with SR-aGVHD.
in the Expanded Access program, Ryoncil was used as a rescue therapy to treat 241 children with SR-aGVHD who failed to receive institutional standard care (steroids and other drugs).
in the Open Label III trial, Ryoncil was also used as a first-line therapy to treat 55 children with SR-aGVHD, 89% of whom were C/D-grade diseases.
in the BLA, Mesoblast also included data on children in the control group of children who received institutional standard care treatment in mount Sinai's Acute GVHD (MAGIC) database for the same period, matching the Open Label III entry criteria and disease severity.
results from the Open Label III study with the MAGIC's contemporaneous control group supported Ryoncil's effectiveness in children with SR-aGVHD, particularly in the most severe childhood patients, and the benefits of survival.
conclusions were supported by the expansion of access to 241 child outcomes in the project.
the data were: 55 children with SR-aGVHD treated with Ryoncil (89% with C/D-grade disease) were treated in the Open Label III study, with a total remission rate of 70% on the 28th day and a survival rate of 75% on the 100th day.
results were better than those of 30 children with SR-aGVHD matched from the MAGIC database over the same period, with a total remission rate of 43% on the 28th day and a survival rate of 57% on the 100th day in the control group.
currently, Ryoncil is evaluating the treatment of a variety of inflammatory diseases, including SR-aGVHD (children, adults), chronic GVHD, biologic refractic Crohn's disease, ischemic ischemic encephalopathy, large herpes skin looseness, and elderly lung disease.
Ryoncil is administered intravenously and is currently evaluating the treatment of a variety of inflammatory diseases, including SR-aGVHD, chronic GVHD, biologic refractic Crohn's disease, ischemic ischemic encephalopathy, and large herpes skin looseness, in patients with elderly lung disease.
the original source: FDA advisory thumbs up on Mesoblast's Ryoncil.