Mesoblast has resubmitted its biologics license application (BLA) to the US Food and Drug Administration (FDA) for approval of remestemcel-L in the treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD).
Survival outcomes have not improved over the past two decades for the most severe forms of SR- aGVHD, a life-threatening complication of an allogeneic bone marrow transplant following treatment for blood cancers and other conditions.
The lack of any approved treatments for children under 12 means that there is an urgent need for a therapy that improves the dismal survival outcomes.
If remestemcel-L receives FDA approval, it will be the first allogeneic “off-the-shelf” cellular medicine to be approved in the United States, and the first therapy for children under 12 years old with SR-aGVHD.
Due to substantial new information, the FDA granted remestemcel-L Fast Track designation, a process to facilitate the development and expedited review of therapies for serious conditions that fill unmet medical needs, and Priority Review designation, given to drugs that treat a serious condition and provide a significant improvement in safety or effectiveness over existing treatments.
The BLA resubmission will have a review period up to six months from filing upon acceptance by the FDA. It contains new long-term survival data of children enrolled in a Phase III trial showing durability of treatment effect through at least four years.
“There is an urgent need for a therapy that improves the dismal survival outcome in children with SR-aGVHD” said Dr Silviu Itescu, Chief Executive of Mesoblast. “Our team has worked tirelessly over the past two years to provide a comprehensive response to the FDA. We are grateful for the agency’s active dialogue and constructive feedback that will ensure a high bar is met in terms of product consistency and predictability of clinical outcomes.”