Cytori Therapeutics, Inc. (NASDAQ: CYTX) announced today that the
European Medicines Agency's Committee for Orphan Medicinal Products
(COMP) issued a positive opinion toward Cytori’s application for an
orphan drug designation for the new Scleroderma therapeutic ECCS-50,
which contains adipose tissue-derived stromal vascular fraction cells
derived from Cytori’s proprietary Celution® System.
Cytori has also recently received FDA approval to study the same
therapeutic, ECCS-50, in an 80 patient Phase 3 pivotal study for
Scleroderma associated hand dysfunction. This trial is currently planned
to begin enrolling this year in approximately 12 U.S. sites.
“The COMP’s supportive opinion is first and foremost a key step toward
receiving orphan designation in the European Union for Cytori Cell
Therapy® in Scleroderma associated hand dysfunction,” said Dr. Marc
Hedrick, President and CEO of Cytori Therapeutics, “In the bigger
picture, this opinion is a validating event for our broader strategy to
achieve disease-specific claims and reimbursement in nearer term
indications, such as those for orphan indications.”
“Orphan designation in Europe comes with significant benefits,” said Ken
Kleinhenz, Cytori Vice President of Global Regulatory and Quality
Affairs. “Companies who obtain orphan designation from the European
Commission benefit from a number of incentives including: protocol
assistance, significant fee reductions, access to the centralized
procedures, and market exclusivity of 10 years once the medicine is on
the market.”
Orphan medicines are eligible to be assessed through the centralized
procedure that allows companies to make a single application to the
European Medicines Agency that would result in a single opinion and a
single decision from the European Commission that would be valid in all
EU Member States. Conditional approval is also available to orphan
medicines through the centralized process. Orphan medicines benefit from
ten years of protection from market competition with similar medicines
with similar indications once they are approved.1
“We have been extremely encouraged by the positive results from our open
label clinical trial evaluating Cytori Cell Therapy (autologous adipose
derived stromal vascular fraction) in patients with impaired hand
function due to Scleroderma. The orphan status should help accelerate
our ability to ultimately provide Cytori Cell Therapy (autologous
adipose derived stromal vascular fraction) to our patients who are truly
in need of additional effective therapies,” said Prof. Brigitte Granel
from the North Hospital, Assistance Publique Hopitaux de Marseilles and
first author of the publication describing the results of the
SCLERADEC–I trial.2
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 orphan designation
for Cytori Cell therapy, and the impact of such designation in
accelerating the availability of Cytori Cell Therapy to treat patients
in Europe, 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 and regulatory 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. 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).
2. Granel B, Dauma A, Jouve E, Harle JR, Nguyen PS, Chabannon C,
Colavolpe N, Reyneir JC, Truillet R, Mallet S, Baiade A, Casanova D,
Giraudo L, Arnaud L, Veran J, Sabatier F, Magalon G. Safety,
tolerability and potential efficacy of injection of autologous
adipose-derived stromal vascular fraction in the fingers of patients
with systemic sclerosis: an open-label phase I trial. Ann Rheum Dis
2014; doe:10.1136/annrheumdis-2014-205681
Copyright Business Wire 2015