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Mesoblast cell therapy gets FDA fast-track tag

March 7, 2017

Mesoblast Limited (Nasdaq:MESO) (ASX:MSB) today announced that the United States Food and Drug Administration (FDA) has granted a Fast Track designation for the use of its cell therapy, MSC-100-IV, to achieve improved overall response rate in children with steroid refractory acute Graft Versus Host Disease (aGVHD).

Fast Track designation has the potential to shorten the time to FDA approval of MSC-100-IV for this indication through priority review (shortened FDA review process from 10 to 6 months) and a streamlined rolling review process (completed sections of the Biologics License Application, BLA, can be submitted for FDA review as they become available, instead of waiting for all to be completed). The product candidate’s existing Orphan Indication designation may additionally lead to potential commercial benefits following FDA approval.

Mesoblast’s application for Fast Track status was supported by the clinical data in 241 pediatric patients with steroid refractory aGVHD who were treated on a single expanded access protocol (EAP) with MSC-100-IV. Overall response rate at Day 28 in this group was 65%, and day 100 survival was significantly improved in children who achieved an overall response at day 28 (82% vs. 39%, log rank p-value <0.0001).

The clinical results from the EAP were also used by Mesoblast in earlier discussions with the FDA that initially established the current accelerated development pathway for MSC-100-IV as front-line therapy in children with steroid-refractory aGVHD. Based on these discussions, Mesoblast believes that a single successful open-label Phase 3 trial will be sufficient for conditional FDA approval.


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