FDA Grants Fast Track Designation for Remestemcel-L in the Treatment of Acute Respiratory Distress Syndrome Due to COVID-19
December 02 2020 - 6:00AM
Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in
allogeneic cellular medicines for inflammatory diseases, today
announced that the United States Food and Drug Administration (FDA)
has granted Fast Track designation for remestemcel-L in the
treatment of acute respiratory distress syndrome (ARDS) due to
COVID-19 infection. Fast Track designation is granted if a therapy
demonstrates the potential to address unmet medical needs for a
serious or life-threatening disease.1 ARDS is the primary cause of
death in patients with COVID-19.
Fast Track designation by the FDA is intended to
facilitate development and expedite review of therapies to treat
serious and life-threatening conditions with no or limited
treatment options so that an approved product can reach the market
expeditiously.1 Under Fast Track designation, a Biologic License
Application (BLA) for remestemcel-L is eligible for both rolling
submission and priority review.
Clinical data provided to the FDA supporting the
potential of remestemcel-L to address the unmet medical need of
COVID-19 ARDS included results from a pilot study of remestemcel-L
under emergency compassionate use at New York’s Mt Sinai Hospital
in March-April this year. In this study, nine of 12
ventilator-dependent patients (75%) with moderate to severe
COVID-19 ARDS were successfully discharged from hospital a median
of 10 days after receiving two intravenous doses of
remestemcel-L.
The ongoing randomized controlled Phase 3 trial of
remestemcel-L in up to 300 ventilator-dependent patients with
moderate to severe COVID-19 ARDS, under an FDA Investigational New
Drug clearance, is approximately two-thirds enrolled. The
trial’s primary endpoint is overall mortality at Day 30, and the
key secondary endpoint is days alive off ventilatory support
through Day 60. Two interim analyses have been performed by the
independent Data Safety Monitoring Board (DSMB), after 90 and 135
patients were enrolled, with recommendations to continue the trial
as planned. A third and final interim analysis is planned to
be performed by the DSMB when 180 patients have completed 30 days
of follow-up.
Mesoblast recently entered into a license and
collaboration agreement with Novartis for the development,
manufacture and commercialization of remestemcel-L, with an initial
focus on the treatment of ARDS, including COVID-19 ARDS. The
closing of the agreement is subject to the expiration or
termination of the waiting period under the Hart-Scott-Rodino
Antitrust Improvements Act and certain other conditions.
About COVID-19 Acute Respiratory Distress
Syndrome (ARDS) ARDS is due to a dysregulated immune
response in the lungs to various infectious agents including
COVID-19. Deaths continue to increase in ventilator-dependent ARDS
patients as COVID-19 cases continue to surge globally. Despite
improved treatment and earlier intervention in hospitalized
COVID-19 patients overall, the mortality rate in COVID-19 ARDS
patients who are over 60 years old remains more than 60%.2 These
patients appear to be particularly refractory to corticosteroids
such as dexamethasone3,4 and have not responded to single cytokine
antagonists, anti-virals, or anti-malaria agents.
About Remestemcel-L Remestemcel-L
is an investigational therapy comprising culture-expanded
mesenchymal stromal cells derived from the bone marrow of an
unrelated donor. Remestemcel-L is thought to have immunomodulatory
properties to counteract the cytokine storms that are implicated in
various inflammatory conditions by downregulating the production of
pro-inflammatory cytokines, increasing production of
anti-inflammatory cytokines, and enabling recruitment of naturally
occurring anti-inflammatory cells to involved tissues.
About Mesoblast Mesoblast Limited
(Nasdaq:MESO; ASX:MSB) is a world leader in developing allogeneic
(off-the-shelf) cellular medicines. The Company has leveraged its
proprietary mesenchymal lineage cell therapy technology platform to
establish a broad portfolio of commercial products and late-stage
product candidates. Mesoblast has a strong and extensive global
intellectual property portfolio with protection extending through
to at least 2040 in all major markets. The Company’s proprietary
manufacturing processes yield industrial-scale, cryopreserved,
off-the-shelf, cellular medicines. These cell therapies, with
defined pharmaceutical release criteria, are planned to be readily
available to patients worldwide. For more information, please see
www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter:
@Mesoblast
References
1.https://www.fda.gov/ForPatients/Approvals/Fast/ucm405399.htm
2.Rezoagli E, Fumagalli R, Bellani G. 2017. Definition and
epidemiology of acute respiratory distress syndrome. Ann Transl
Med. 5(14): 1- 12 3.Bellani G, Laffey J, Pham T. et. al. 2016.
Epidemiology, patterns of care, and mortality for patients with
acute respiratory distress syndrome in intensive care units in 50
countries. JAMA 315(8): 788-800
4.https://www.hopkinsguides.com/hopkins/view/Johns_Hopkins_ABX_Guide/540747/all/Coronavirus_COVID_19__SARS_CoV_2
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Release authorized by the Chief Executive.
For further information, please contact:
Media Julie Meldrum T: +61 3 9639 6036
E:julie.meldrum@mesoblast.com |
Kristen Bothwell T: +1 917 613 5434
E:kbothwell@rubenstein.com |
Investors Schond Greenway T: +212 880 2060
E: schond.greenway@mesoblast.com |
Paul Hughes
T: +61 3 9639 6036 E: paul.hughes@mesoblast.com |
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