TiGenix granted Orphan Drug Designation from the U.S. FDA for Cx601
(Thomson Reuters ONE) -
PRESS RELEASE
TiGenix granted Orphan Drug Designation from the U.S. FDA for Cx601
Leuven (BELGIUM) - October 23, 2017, 7:00h CEST - TiGenix NV (Euronext Brussels
and NASDAQ: TIG), an advanced biopharmaceutical company focused on exploiting
the anti-inflammatory properties of allogeneic, or donor-derived, stem cells to
develop novel therapies for serious medical conditions, today announces that the
U.S. Food and Drug Administration (FDA) has granted orphan drug designation
(ODD) to Cx601 for the treatment of patients with fistulizing Crohns disease.
TiGenix started a global pivotal Phase III clinical trial in the first half of
2017 with the aim of submitting a future U.S. Biologics License Application
(BLA) to the FDA for Cx601, a first-in-class allogeneic cell therapy product for
the treatment of complex perianal fistulas in patients with Crohn's disease who
have had an inadequate response to at least one conventional or biologic
therapy. In parallel, TiGenix is exploring expedited pathways to accelerate the
submission and review process for U.S. regulatory approval.
"The granting of orphan drug status by the FDA is a significant step forward in
the Cx601 development program" said Dr. María Pascual, Vice President Regulatory
Affairs and Corporate Quality at TiGenix. "The FDA's recognition of Cx601 as an
orphan drug brings a number of potential financial benefits and is aligned with
our ongoing work seeking expedited pathways towards product approval in the
U.S."
The FDA grants orphan status for novel products to treat conditions affecting
fewer than 200,000 people in the U.S. Orphan designation, which is intended to
facilitate drug development for rare diseases, provides substantial benefits to
the sponsor, including seven years of market exclusivity following marketing
approval, tax credits for clinical research costs, eligibility for orphan
product grants and the waiver of certain administrative fees.
For more information please contact:
Claudia Jiménez
Senior Director Investor Relations and Communications
Tel: +34918049264
Claudia.jimenez(at)tigenix.com
About TiGenix
TiGenix NV (Euronext Brussels and NASDAQ: TIG) is an advanced biopharmaceutical
company developing novel therapies for serious medical conditions by exploiting
the anti-inflammatory properties of allogeneic, or donor-derived, stem cells.
TiGenix lead product, Cx601, has successfully completed a European Phase III
clinical trial for the treatment of complex perianal fistulas - a severe,
debilitating complication of Crohn's disease. Cx601 has been filed for
regulatory approval in Europe and a global Phase III trial intended to support a
future U.S. Biologic License Application (BLA) started in 2017. TiGenix has
entered into a licensing agreement with Takeda, a global pharmaceutical company
active in gastroenterology, under which Takeda acquired the exclusive right to
develop and commercialize Cx601 for complex perianal fistulas outside the U.S.
TiGenix' second adipose-derived product, Cx611, is undergoing a Phase I/II trial
in severe sepsis - a major cause of mortality in the developed world. Finally,
AlloCSC-01, targeting acute ischemic heart disease, has demonstrated positive
results in a Phase I/II trial in acute myocardial infarction (AMI). TiGenix is
headquartered in Leuven (Belgium) and has operations in Madrid (Spain) and
Cambridge, MA (USA). For more information, please visit http://www.tigenix.com.
About Cx601
Cx601 is a local administration of allogeneic (or donor derived) expanded
adipose-derived stem cells (eASCs) for the treatment of complex perianal
fistulas in Crohn's disease patients that have previously failed conventional
therapy. Crohn's disease is a chronic inflammatory disease of the intestine and
complex perianal fistulas are a severe and debilitating complication for which
there is currently no effective treatment. Cx601 was granted orphan drug
designation by the European Commission in 2009. TiGenix completed a European
Phase III clinical trial (ADMIRE-CD) in August 2015. The 24-week data were
published in the Lancet and showed both the primary endpoint and the safety and
efficacy profile were met[1]. A follow-up analysis was completed at 52 weeks and
104 weeks post-treatment, confirming the sustained efficacy and safety profile
of the product[2]. The 24-week results of the Phase III ADMIRE-CD trial were
published in The Lancet in July 2016.1 Based on the positive 24 weeks Phase III
study results, TiGenix submitted a Marketing Authorization Application to the
European Medicines Agency (EMA) and a CHMP opinion is expected in 2017. A global
Phase III clinical trial (ADMIRE-CD II) intended to support a future U.S.
Biologic License Application (BLA) started in 2017, based on a trial protocol
that has been agreed with the Food and Drug Administration (FDA) through a
special protocol assessment procedure (SPA) (clinicaltrials.gov; NCT03279081).
ADMIRE-CD II is a randomized, double-blind, placebo-controlled study designed to
confirm the efficacy and safety of a single administration of Cx601 for the
treatment of complex perianal fistulas in Crohn's disease patients. In July
2016, TiGenix entered into a licensing agreement with Takeda, a global
pharmaceutical company active in gastroenterology, under which Takeda acquired
exclusive rights to develop and commercialize Cx601 for complex perianal
fistulas in Crohn's patients outside of the U.S.
Forward-looking information
This press release may contain forward-looking statements and estimates with
respect to the anticipated future performance of TiGenix and the market in which
it operates. Certain of these statements, forecasts and estimates can be
recognised by the use of words such as, without limitation, "believes",
"anticipates", "expects", "intends", "plans", "seeks", "estimates", "may",
"will" and "continue" and similar expressions. They include all matters that are
not historical facts. Such statements, forecasts and estimates are based on
various assumptions and assessments of known and unknown risks, uncertainties
and other factors, which were deemed reasonable when made but may or may not
prove to be correct. Actual events are difficult to predict and may depend upon
factors that are beyond the Company's control. Therefore, actual results, the
financial condition, performance or achievements of TiGenix, or industry
results, may turn out to be materially different from any future results,
performance or achievements expressed or implied by such statements, forecasts
and estimates. Given these uncertainties, no representations are made as to the
accuracy or fairness of such forward-looking statements, forecasts and
estimates. Furthermore, forward-looking statements, forecasts and estimates only
speak as of the date of the publication of this press release. TiGenix disclaims
any obligation to update any such forward-looking statement, forecast or
estimates to reflect any change in the Company's expectations with regard
thereto, or any change in events, conditions or circumstances on which any such
statement, forecast or estimate is based, except to the extent required by
Belgian law.
--------------------------------------------------------------------------------
[1] Panés J, García-Olmo D, Van Assche G et al., Expanded allogeneic adipose-
derived mesenchymal stem cells (Cx601) for complex perianal fistulas in Crohn's
disease: a phase 3 randomized, double-blind controlled trial. The Lancet.
2016; 388(10051):1281-90.
[2] Panes, J. et al., OP009 Long-term efficacy and safety of Cx601, allogeneic
expanded adipose-derived mesenchymal stem cells, for complex perianal fistulas
in Crohn's disease: 52-week results of a phase III randomised controlled trial.
J Crohn's Colitis. 2017; 11: S5-S5
This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: TiGenix via GlobeNewswire
Unternehmensinformation / Kurzprofil:
Datum: 23.10.2017 - 07:00 Uhr
Sprache: Deutsch
News-ID 564651
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contact information:
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