MSC-100-IV Receives Fast-Track Designation

The U.S. Food and Drug Administration has granted fast-track designation for MSC-100-IV for pediatric patients with steroid-refractory acute graft-versus-host disease (aGVHD) following allogeneic hematopoietic cell transplantation.

The decision was supported by clinical data from a study of 241 pediatric patients with steroid-refractory aGVHD treated with MSC-100-IV (an intravenously delivered product comprising 100 million mesenchymal stem cells per unit). By day 28, the overall response rate was 65 percent, thus significantly improving day 100 survival in children in this group (82% vs. 39%, p<0.0001).

MSC-100-IV was previously granted orphan drug designation. The drug’s manufacturers are planning an open-label, phase III trial for children with steroid-refractory aGVHD that will begin at the end of 2017.

Source: Mesoblast press release, March 7, 2017.