FDA ACCEPTS MESOBLAST’S BIOLOGICS LICENCE APPLICATION FOR RYONCIL™ AND AGREES TO PRIORITY REVIEW
April 01 2020 - 6:00AM
Mesoblast Limited (Nasdaq: MESO; ASX:MSB), global leader in
cellular medicines for inflammatory diseases, today announced that
the United States Food and Drug Administration (FDA) has accepted
for priority review the Company’s Biologics License Application
(BLA) filing for RYONCILTM (remestemcel-L), its allogeneic cell
therapy for the treatment of children with steroid-refractory acute
graft versus host disease (SR-aGVHD). The FDA has set a
Prescription Drug User Fee Act (PDUFA) action date of September 30,
2020, and if approved, Mesoblast will make RYONCIL immediately
available in the United States.
A Priority Review designation will direct overall
attention and resources to the evaluation of applications for drugs
that, if approved, would be significant improvements in the safety
or effectiveness of the treatment, diagnosis, or prevention of
serious conditions when compared to standard applications. The FDA
has advised that they are planning to hold an Advisory Committee
Meeting to discuss this application.
Mesoblast Chief Executive Dr Silviu Itescu stated:
“There is a critical need to improve survival outcomes in children
suffering from the more advanced stages of this devastating
disease. The acceptance of the BLA represents an important
milestone for the Company. Mesoblast is on track in its preparation
for the potential launch of RYONCIL, including meeting its target
inventory build and commercial team roll-out.”
About Acute GVHD Acute GVHD occurs in
approximately 50% of patients who receive an allogeneic bone marrow
transplant (BMT). Over 30,000 patients worldwide undergo an
allogeneic BMT annually, primarily during treatment for blood
cancers, and these numbers are increasing.1 In patients with the
most severe form of acute GVHD (Grade C/D or III/IV) mortality is
as high as 90% despite optimal institutional standard of care.2,3.
There are currently no FDA-approved treatments in the US for
children under 12 with SR-aGVHD.
About RYONCILTM Mesoblast’s lead product
candidate, RYONCIL (remestemcel-L), is an investigational therapy
comprising culture- expanded mesenchymal stem cells derived from
the bone marrow of an unrelated donor. It is administered to
patients in a series of intravenous infusions. RYONCIL is believed
to have immunomodulatory properties to counteract the inflammatory
processes that are implicated in SR- aGVHD by down-regulating the
production of pro-inflammatory cytokines, increasing production of
anti-inflammatory cytokines, and enabling recruitment of naturally
occurring anti-inflammatory cells to involved tissues.
References
- Niederwieser D, Baldomero H, Szer J. (2016)
Hematopoietic stem cell transplantation activity worldwide in 2012
and a SWOT analysis of the Worldwide Network for Blood and Marrow
Transplantation Group including the global survey.
- Westin, J., Saliba, RM., Lima, M. (2011) Steroid-refractory
acute GVHD: predictors and outcomes. Advances in Hematology.
- Axt L, Naumann A, Toennies J (2019) Retrospective single center
analysis of outcome, risk factors and therapy in steroid refractory
graft-versus-host disease after allogeneic hematopoietic cell
transplantation. Bone Marrow Transplantation.
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’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.
Mesoblast has filed a Biologics License Application to
the United States Food and Drug Administration (FDA) to
seek approval of its product candidate RYONCIL™ (remestemcel-L) for
steroid-refractory acute graft versus host disease (acute GvHD).
Remestemcel-L is also being developed for other rare diseases.
Mesoblast is completing Phase 3 trials for its product candidates
for advanced heart failure and chronic low back pain. If approved,
RYONCIL is expected to be launched in the United
States in 2020 for pediatric steroid-refractory acute GVHD.
Two products have been commercialized
in Japan and Europe by Mesoblast’s licensees,
and the Company has established commercial partnerships
in Europe and China for certain Phase 3
assets.
Mesoblast has a strong and extensive global intellectual
property (IP) portfolio with protection extending through to at
least 2040 in all major markets. This IP position is expected to
provide the Company with substantial commercial advantages as it
develops its product candidates for these conditions.
Mesoblast has locations in Australia, the United
States and Singapore and is listed on
the Australian Securities Exchange (MSB) and on the
Nasdaq (MESO). For more information, please
see www.mesoblast.com, LinkedIn: Mesoblast
Limited and Twitter: @Mesoblast
Forward-Looking Statements This announcement
includes forward-looking statements that relate to future events or
our future financial performance and involve known and unknown
risks, uncertainties and other factors that may cause our actual
results, levels of activity, performance or achievements to differ
materially from any future results, levels of activity, performance
or achievements expressed or implied by these forward-looking
statements. We make such forward-looking statements pursuant to the
safe harbor provisions of the Private Securities Litigation Reform
Act of 1995 and other federal securities laws. Forward- looking
statements should not be read as a guarantee of future performance
or results, and actual results may differ from the results
anticipated in these forward-looking statements, and the
differences may be material and adverse. Forward-looking statements
include, but are not limited to, statements about the timing,
progress and results of Mesoblast’s preclinical and clinical
studies; Mesoblast’s ability to advance product candidates into,
enroll and successfully complete, clinical studies; the timing or
likelihood of regulatory filings and approvals; and the pricing and
reimbursement of Mesoblast’s product candidates, if approved. You
should read this press release together with our risk factors, in
our most recently filed reports with the SEC or on our website.
Uncertainties and risks that may cause Mesoblast’s actual results,
performance or achievements to be materially different from those
which may be expressed or implied by such statements, and
accordingly, you should not place undue reliance on these
forward-looking statements. We do not undertake any obligations to
publicly update or revise any forward-looking statements, whether
as a result of new information, future developments or
otherwise.
Release authorized by the Chief Executive.
For further information, please contact: Julie Meldrum
Corporate Communications
T: +61 3 9639 6036
E: julie.meldrum@mesoblast.com
Schond Greenway Investor RelationsT: +1 212 880 2060E:
schond.greenway@mesoblast.com
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