FDA Grants Fast Track Designation for Mesoblast’s Cell Therapy in Children With Acute Graft Versus Host Disease
March 07 2017 - 6:00AM
Mesoblast Limited (Nasdaq:MESO) (ASX:MSB) today announced that
the United States Food and Drug Administration (FDA) has granted a
Fast Track designation for the use of its cell therapy, MSC-100-IV,
to achieve improved overall response rate in children with steroid
refractory acute Graft Versus Host Disease (aGVHD).
Fast Track designation has the potential to
shorten the time to FDA approval of MSC-100-IV for this indication
through priority review (shortened FDA review process from 10 to 6
months) and a streamlined rolling review process (completed
sections of the Biologics License Application, BLA, can be
submitted for FDA review as they become available, instead of
waiting for all to be completed). The product candidate's
existing Orphan Indication designation may additionally lead
to potential commercial benefits following FDA approval.
Mesoblast’s application for Fast Track status
was supported by the clinical data in 241 pediatric patients with
steroid refractory aGVHD who were treated on a single expanded
access protocol (EAP) with MSC-100-IV. Overall response rate at Day
28 in this group was 65%, and day 100 survival was significantly
improved in children who achieved an overall response at day 28
(82% vs. 39%, log rank p-value <0.0001).
The clinical results from the EAP were also used
by Mesoblast in earlier discussions with the FDA that initially
established the current accelerated development pathway for
MSC-100-IV as front-line therapy in children with
steroid-refractory aGVHD. Based on these discussions,
Mesoblast believes that a single successful open-label Phase 3
trial will be sufficient for conditional FDA approval.
In November 2016, Mesoblast reported that the
ongoing 60-patient open label Phase 3 registration trial of
MSC-100-IV in children with steroid refractory aGVHD was
successful in a pre-specified interim futility analysis
using the trial’s primary endpoint of Day 28 overall
responses. The futility threshold was established using a
Bayesian analysis method which determined the likelihood of
obtaining a statistically significant treatment effect at study
completion based on the data observed at this interim time
point. Enrollment in this Phase 3 trial is expected to
complete mid-2017 with a top-line read out in 2H CY 2017.
In December 2016, Mesoblast entered into
exclusive negotiations with Mallinckrodt Pharmaceuticals for a
commercial and development partnership to develop product
candidates for pediatric and adult aGVHD, outside of Japan and
China. MSC-100-IV is marketed as TEMCELL HS Inj.® for
acute GVHD in children and adults in Japan by Mesoblast’s licensee,
JCR Pharmaceuticals.
About MesoblastMesoblast
Limited (Nasdaq:MESO) (ASX:MSB) is a global leader in developing
innovative cell-based medicines. The Company has leveraged its
proprietary technology platform, which is based on specialized
cells known as mesenchymal lineage adult stem cells, to establish a
broad portfolio of late-stage product candidates. Mesoblast’s
allogeneic, ‘off-the-shelf’ cell product candidates target advanced
stages of diseases with high, unmet medical needs including
cardiovascular diseases, immune-mediated and inflammatory
disorders, orthopedic disorders, and oncologic/hematologic
conditions.
Forward-Looking StatementsThis
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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,
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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
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the differences may be material and adverse. 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.
For further information, please contact:
Julie Meldrum
Corporate Communications
Mesoblast
T: +61 3 9639 6036
E: julie.meldrum@mesoblast.com
Schond Greenway
Investor Relations
Mesoblast
T: +1 212 880 2060
E: schond.greenway@mesoblast.com
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