105 0 Kommentare Appendix 4c Quarterly Activity Report for Quarter Ended March 31, 2024

FDA Provides Clarity on Path to Licensure for Remestemcel-L in SR-aGVHD and Rexlemestrocel-L in End-Stage Heart Failure

NEW YORK, April 29, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an activity report for the third quarter ended March 31, 2024.

Mesoblast Chief Executive Silviu Itescu said: “We are very pleased with the positive interactions we had last quarter with the FDA, having received clarity on the path to licensure for our product candidates in pediatric acute graft versus host disease and in ischemic patients with chronic heart failure.”

“Based on the clear responses and guidance from FDA we intend this quarter to resubmit our Biologics License Application (BLA) for approval of remestemcel-L in children with SR-aGVHD,” said Mesoblast CEO Dr. Silviu Itescu. “In addition, FDA informed us that the results from our pivotal study of rexlemestrocel-L in end-stage heart failure patients may support an accelerated approval, and we intend to have a pre-BLA meeting to discuss the data that will be provided and the timing for an accelerated approval filing.”

ACTIVITY REPORT

Graft versus Host Disease – Pediatric and Adult Indications

United States Food and Drug Administration (FDA) informed Mesoblast after its Type C meeting during the quarter that following additional consideration the available clinical data from its Phase 3 study MSB-GVHD001 appears sufficient to support submission of the proposed Biologics License Application (BLA) for remestemcel-L for treatment of pediatric patients with steroid-refractory acute graft versus host disease (SR-aGVHD).
Mesoblast intends to file the resubmission this quarter, potentially resulting in an approval for Ryoncil (remestemcel-L) in the second half of CY2024.
Mesoblast will now focus on its original strategy to first gain pediatric approval for RYONCIL, followed by label extension in the larger adult population.

Cardiovascular – Chronic Heart Failure with Reduced Ejection Fraction (HFrEF) in Adults

FDA informed the Company in formal minutes following the Type B meeting held in February under Mesoblast’s existing Regenerative Medicine Advanced Therapy (RMAT) designation, that it supports an accelerated approval pathway for Revascor (rexlemestrocel-L), Mesoblast’s allogeneic mesenchymal precursor cell (MPC) product, in patients with end-stage ischemic heart failure with reduced ejection fraction (HFrEF) and a left ventricular assist device (LVAD).
In these patients, a single administration of REVASCOR reduced inflammation, strengthened left ventricular function, reduced right ventricular failure, reduced hospitalizations and reduced mortality.
REVASCOR has also shown the potential to reduce major adverse cardiac events (MACE) such as cardiovascular death, heart attacks and strokes in ischemic HFrEF patients with NYHA class II /III disease and inflammation.
Mesoblast intends to request a pre-BLA meeting with FDA to discuss data presentation, timing and FDA expectations for an accelerated approval filing in ischemic HFrEF patients with end-stage heart failure.