Ann: FDA Schedules GvHD Pre-License Manufacturing Inspection, page-11

FDA SCHEDULES PRE-LICENSE INSPECTION OF REMESTEMCEL-L MANUFACTURING
Biologics License Application (BLA) Resubmission for Pediatric Acute Graft-Versus-Host
Disease (SR-aGVHD) Currently Being Reviewed
PDUFA Goal Date August 2, 2023
If Approved, Remestemcel-L will be the First Allogeneic “Off-the-Shelf” Cellular Medicine in
the US, and the First Therapy for Children Under 12 Years Old with SR-aGVHD
Melbourne, Australia; March 22 and New York, USA; March 21, 2023: Mesoblast Limited
(ASX:MSB; Nasdaq:MESO), global leader in allogeneic cellular medicines for inflammatory diseases,
today announced that, as part of the ongoing review of the BLA for remestemcel-L in the treatment of
children with SR-aGVHD, the United States Food and Drug Administration (FDA) has scheduled a PreLicense Inspection (PLI) of Mesoblast’s cell therapy manufacturing operations at Lonza Bioscience in
Singapore.
On March 7, 2023, the FDA accepted the Company’s resubmission of the BLA for remestemcel-L and set
a Prescription Drug User Fee Act (PDUFA) goal date of August 2, 2023.
Remestemcel-L has FDA 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,
which is given to drugs that treat a serious condition and provide a significant improvement in safety or
effectiveness over existing treatments. Survival outcomes have not improved over the past two decades
for children or adults with the most severe forms of SR-aGVHD.1-3 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 in children.
“We are pleased that the pre-license inspection of our manufacturing operations has been scheduled.
We look forward to continuing to work closely with FDA during the review period with the aim to make
remestemcel-L available as a therapy for children suffering from SR-aGVHD,” said Mesoblast Chief
Executive Silviu Itescu.