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FDA grants fast track designation to MSC-100-IV for acute GVHD in children

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The FDA granted fast track designation to MSC-100-IV for the treatment of children with steroid-refractory acute graft-versus-host disease following bone marrow transplantation, according to the drug’s manufacturer.

MSC-100-IV (Mesoblast) is comprised of 100 million mesenchymal stem cells per unit dose and is administered intravenously.

The application for fast track designation included clinical data from 241 children with steroid-refractory acute graft-versus-host disease (GVHD) who were treated on a single expanded access protocol with MSC-100-IV.

Sixty-five percent of patients achieved an overall response at day 28 of follow-up. The rate of 100-day survival was significantly higher in those who had achieved a response (82% vs. 39%; P < .0001).

An ongoing open label phase 3 registration trial designed to evaluate MSC-100-IV in 60 children with steroid-refractory acute GVHD was successful in a prespecified interim futility analysis using the trial’s primary endpoint of ORR at day 28.

Enrollment of this trial and its top-line results are expected this year, a company-issued release said.