Melbourne, Australia; October 2, 2020 and New York, USA; October 1, 2020: Mesoblast
Limited (ASX:MSB; Nasdaq:MESO), global leader in allogeneic cellular medicines for inflammatory
diseases, announced today that the US Food and Drug Administration (FDA) has issued a Complete
Response Letter to its Biologics License Application (BLA) for remestemcel-L for the treatment of
pediatric steroid-refractory acute graft versus host disease (SR-aGVHD). While the Oncologic Drugs
Advisory Committee (ODAC)1 of the FDA voted 9:1 that the available data support the efficacy of
remestemcel-L in pediatric patients with SR-aGVHD, the FDA recommended that Mesoblast conduct at
least one additional randomized, controlled study in adults and/or children to provide further evidence
of the effectiveness of remestemcel-L for SR-aGVHD. As there are currently no approved treatments
for this life-threatening condition in children under 12, Mesoblast will urgently request a Type A
meeting with the FDA, expected within 30 days, to discuss a potential accelerated approval with a
post-approval condition for an additional study.
Joanne Kurtzberg, MD, Jerome Harris Distinguished Professor of Pediatrics, Director, Pediatric Blood
and Marrow Transplant Program, and Co-Director, Stem Cell Transplant Laboratory Duke University
Medical Center, said: “The Phase 3 trial results showed that remestemcel-L provides a meaningful
treatment for children with SR-aGVHD who have a very dismal prognosis. I look forward to having this
much-needed therapy available to our patients.”
Mesoblast is currently conducting a randomized, controlled Phase 3 trial evaluating remestemcel-L in
up to 300 ventilator-dependent adults with moderate to severe acute respiratory distress syndrome
(ARDS) due to COVID-19. A second interim analysis by the trial’s independent Data Safety Monitoring
Board is expected in early November, with completion of patient enrollment expected in December.
COVID-19 ARDS is an inflammatory disease with a similar profile of damaging inflammatory cytokines
as is seen in children with SR-aGVHD, and is the primary cause of death in COVID-19 infection. The
trial’s primary endpoint is reduction of all-cause mortality within 30 days of randomization.
The FDA also identified a need for further scientific rationale to demonstrate the relationship of
potency measurements to the product’s biologic activity. Assays measuring the potency of
remestemcel-L will continue to be refined to provide further scientific rationale for its use in severe
inflammatory diseases with high mortality risk, such as SR-aGVHD and COVID-19 ARDS.
Mesoblast Chief Executive Dr Silviu Itescu stated: “We are working tirelessly to bring remestemcel-L
to patients with life threatening inflammatory conditions, including SR-aGVHD and COVID-19 ARDS.”
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