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Cytori Receives Positive European Opinion on Orphan Drug Status
Source: Business Wire
Cytori Therapeutics, Inc. (NASDAQ: CYTX) announced today that the European Medicines Agency's Committee for Orphan Medicinal Products (COMP) issued a positive opinion toward Cytoris application for an orphan drug designation for the new Scleroderma therapeutic ECCS-50, which contains adipose tissue-derived stromal vascular fraction cells derived from Cytoris 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 COMPs 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 SCLERADECI 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 Cytoris 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 (eur-lex.europa.eu/LexUriServ/...: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
Source: Business Wire
Cytori Therapeutics, Inc. (NASDAQ: CYTX) announced today that the European Medicines Agency's Committee for Orphan Medicinal Products (COMP) issued a positive opinion toward Cytoris application for an orphan drug designation for the new Scleroderma therapeutic ECCS-50, which contains adipose tissue-derived stromal vascular fraction cells derived from Cytoris 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 COMPs 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 SCLERADECI 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 Cytoris 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 (eur-lex.europa.eu/LexUriServ/...: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
