Cytori Granted Orphan Drug Status for Cellular Therapeutic in European Union
07 April 2015 - 2:00PM
Business Wire
Cytori Therapeutics, Inc. (NASDAQ: CYTX) announced today that
the European Commission, acting on the positive recommendation from
the European Medicines Agency Committee for Orphan Medicinal
Products, has designated Cytori’s ECCS-50 cellular therapeutic as
an orphan medicinal product for the treatment of scleroderma. This
designation marks the first autologous adipose derived cell therapy
for scleroderma granted orphan status in the European Union
(EU).
Cytori is currently working with leading scleroderma centers in
France led by the Assistance Publique des Hôpitaux de Marseille to
begin a 40 patient EU pivotal trial called SCLERADEC-II. The trial
is a follow-up controlled trial to the first SCLERADEC trial
published last August (Granel et al. Annals of the Rheumatic
Diseases, 2014). If successful, this trial would potentially lead
to EMA approval of Cytori’s lead therapeutic for this indication in
the EU, as an ATMP or Advanced Therapy Medicinal Product.
“Obtaining orphan drug designation in the EU, like FDA Phase III
pivotal trial approval for the same indication in the US, is an
important milestone and part of our corporate strategy of targeting
rare or niche indications with clear unmet medical needs that can
be brought to market rapidly,” said Dr. Marc Hedrick, President and
CEO of Cytori Therapeutics. “Orphan approval gives Cytori a number
of advantages that greatly facilitates us getting a novel drug into
a market with favorable reimbursement dynamics.”
Cytori recently received FDA approval for a phase 3 clinical
trial with our ECCS-50 therapeutic in 80 patients with scleroderma
associated hand dysfunction. This US clinical trial is currently
planned to begin enrolling this year and has recently been granted
FDA approval to expand the number of centers from 12 to 20 sites.
This US clinical data could also be useful to support regulatory
approvals and reimbursement in the EU as well as other global
markets.
The European Commission grants orphan designations for medicines
that treat a life-threatening or chronically debilitating condition
affecting no more than five in 10,000 persons in the EU and where
no satisfactory treatment is available. In Europe, the scleroderma
affected population appears to be between 82/million1,2 and
280/million4, which would account for a market size of 61,000 to
208,000 of scleroderma patients. Scleroderma is estimated to have a
prevalence of 240/million in the adult population which equates to
approximately 50,000 scleroderma people in the United States3.
“Scleroderma involves the hands in nearly 90% of patients and
nearly always is accompanied by pain and impairment of simple daily
activities that we all take for granted,” said Dr. Steven Kesten,
Chief Medical Officer of Cytori Therapeutics. “Hand symptoms are
the leading cause of disability in this patient population, more
disabling than rheumatoid arthritis, and severely negatively impact
quality of life. In addition to general measures to protect the
hand, patients require medications, which often are inadequate to
control symptoms or prevent complications. As our
fingers and hand are so critical in everyday activities, people
with scleroderma are constantly aware of their
disease. We are encouraged by the data generated to date
and believe that the prospective trials in the US and France will
provide the level of evidence needed to definitely demonstrate the
benefit of Cytori Cell Therapy."
“A significant portion of the overall European cell therapy
approval process has now been completed given our existing CE Mark
on the Celution System”, stated Kenneth Kleinhenz, Vice President
of Regulatory Affairs and Quality Assurance at Cytori Therapeutics.
“All cell therapy products used in combination with a medical
device require external review by a European notified body. The CE
Mark certificate previously issued by our notified body, achieved
this requirement and includes much of the required manufacturing
requirements since the ECCS-50 drug product is manufactured
exclusively with the Celution System at the point of care. This
modular approach alleviates many of the regulatory uncertainties
with the manufacturing aspects of the EMA approval process, leaving
the clinical data as the remaining milestone required for market
authorization.”
In addition, a range of development, regulatory, and financial
incentives are offered in the EU for medicines that have been
granted an orphan designation by the European Commission5:
- A form of scientific advice called
protocol assistance
- Access to the centralized authorization
procedure using a single application
- Additional incentives for micro, small,
and medium-sized enterprises including administrative and
procedural assistance and fee reductions
- 10 years of market exclusivity once
approved
- Reduced fees for regulatory
activities
- Incentives in EU member states
About Cytori Therapeutics, Inc.
Cytori Therapeutics is a late stage cell therapy company
developing autologous cell therapies from adipose tissue to treat a
variety of medical conditions. Data from preclinical studies and
clinical trials suggest that Cytori Cell Therapy™ acts principally
by improving blood flow, modulating the immune system, and
facilitating wound repair. As a result, Cytori Cell Therapy™ may
provide benefits across multiple disease states and can be made
available to the physician and patient at the point-of-care through
Cytori’s proprietary technologies and products. For more
information: visit www.cytori.com.
Cautionary Statement Regarding Forward-Looking Statements
This communication includes forward-looking statements regarding
events, trends and business prospects, which may affect our future
operating results and financial position. Such statements,
including, but not limited to, those regarding our ability to
achieve a marketing authorization for Cytori Cell Therapy™, and the
impact of a CE Mark on the Celution System in accelerating the
European marketing approval, are forward looking statements. Such
statements are subject to risks and uncertainties that could cause
our actual results and financial position to differ materially.
Some of these risks include clinical, regulatory and reimbursement
uncertainties, the challenges inherent in convincing physicians and
patients to adopt the new technology, dependence on third party
performance, performance and acceptance of our products, and other
risks and uncertainties described under the “Risk Factors” in
Cytori's Securities and Exchange Commission Filings,
including in its most recent annual and quarterly reports. Cytori
assumes no responsibility to update or revise any forward-looking
statements contained in this press release to reflect events,
trends or circumstances after the date of this communication.
1. Rheumatology (Oxford). 2004 May;43(5):596-602. Epub 2004 Feb
10.
2. Medicine (Baltimore). 2008 Sep;87(5):272-80. doi:
10.1097/MD.0b013e318189372f.
3. Rheum Dis Clin North Am. 2003 May;29(2):239-5
4. Rheumatology (Oxford). 2004 Sep;43(9):1129-37. Epub 2004 Jun
22.
5. Regulation (EC) No 141/2000 on orphan medicinal products
(http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2000:018:0001:0005:en:PDF).
Cytori Therapeutics, Inc.Shawn Richardson,
1-858-875-5279ir@cytori.com
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