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TOPIC: Final EMA orphan disease designation

Final EMA orphan disease designation 07 Apr 2015 08:08 #4090

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Relatively fast...

SAN DIEGO--(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 Commission 5:

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

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Board moderator and Site-owner. I still regret the day I started analysing the prospects of MacroPore (now Cytori) back in 2004- a left-over from the tech-bubble at that time from the century change in my portfolio- and became addicted to Cytori´s fat cell technology. :cry:

Final EMA orphan disease designation 07 Apr 2015 08:30 #4091

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I hope that today the 1.42 fall

holdi

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holdi

Final EMA orphan disease designation 07 Apr 2015 08:45 #4092

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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.


The word AMTP is a red flag for me, but being able to reach this designation and marketing authorization with 40 patients is astonishing and of great value. :vegas:

“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.”


If I get this straight- existing device claims PLUS some clinical data from apps passing the central EMA office GIVES central approval?

Worth the trouble and maybe the regulatory path for several apps???? :grin: :KO:

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Board moderator and Site-owner. I still regret the day I started analysing the prospects of MacroPore (now Cytori) back in 2004- a left-over from the tech-bubble at that time from the century change in my portfolio- and became addicted to Cytori´s fat cell technology. :cry:

Final EMA orphan disease designation 07 Apr 2015 08:54 #4093

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All good news today/yesterday.........technically nothing means nothing until we get 2 consecutive daily closes above 142. Then it becomes MAJOR support once again. There will be enough count to et us back to the old high after this........10 bucks per share .........then , when you get 2 consec closes above the all time high , there will be enough count to take us to 75 dollars per share ........it will go there without a buyout.......lots of stuff between here and there....... :cool: forgot
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Final EMA orphan disease designation 07 Apr 2015 09:31 #4095

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Forgot,your smokin the good stuff today. Fas, hard to get excited about anything that happens in Europe as we have many approvals in Europe with absolutely nothinng to show for it revenue wise. I just can't give these guys the benefit of the doubt anymore. If Cytori were in better management hands our pps would not sit at these pathetic levels and we would not have given the company away. Many more warrants to be converted at .57. We still have done zero to correct the balance sheet,so the #1 concern continues to be the elephant in the room. Any other company with this technology would be at $50 already with partnerships galore but here we sit broken hearted. Anyone bother to check out the Lorem Vascular website which is still non operational. Just sayin, you can't make this stuff up. :bang:

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