Mesoblast (ASX:MSB) met with the US FDA watchdog for skin therapies to discuss their letter for a cell treatment to address steroid-refractory acute graft versus host (GVHD) disease in children.
Currently there are no therapies to treat GVHD in children under the age of 12 with the disease described as a severe inflammation in the bloodstream caused by complications of bone marrow transplants.
In 30,000 patients each year, the disease can occur in up to 50 per cent of cases who receive a transplant primarily during treatment for blood cancers, with death as high as 90 per cent for severe cases.
The US Food and Drug Administration’s Office of Tissues and Advanced Therapies indicated that Mesoblast’s approach to address the outstanding chemistry, manufacturing and controls items are reasonable.
They also outlined that the in vitro immunomodulatory activity Mesoblast intends to measure for potency is a reasonable critical quality attribute for the product, and the relevance of this activity to clinical outcomes should be established.
Mesoblast has now generated substantial new data that it believes establish the relevance of the proposed in vitro immunomodulatory activity of remestemcel-L to the in vivo clinical effect of the product in the Phase 3 trial in children with steroid-refractory acute graft versus host, including survival and biomarkers of in vivo activity.
They company is slated to provide this new data to the US FDA watchdog for skin therapies and address other outstanding items required to resubmit its Biologics license application.
Shares in Mesoblast (ASX:MSB) are trading 2.9 per cent higher at $1.40.