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Cytori Receives Positive European Opinion on Orphan Drug Status

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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

Posted-In: News FDA Press Releases


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