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TOPIC: Orphan drug status for Scleroderma in EU

Orphan drug status for Scleroderma in EU 24 Feb 2015 07:48 #3483

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Cytori Therapeutics, Inc. (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.

“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


Nice- was a no brainer, but a surprise that they applied after all. :grin: :cool:
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Orphan drug status for Scleroderma in EU 24 Feb 2015 07:56 #3485

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Fas, Do you have any insight as to next step/steps? How long before approval?

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Orphan drug status for Scleroderma in EU 24 Feb 2015 08:16 #3487

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months.

Fas, Do you have any insight as to next step/steps? How long before approval?


My insight is limited to the fact that Scleradec II is a pivotal device trial, which would bring it on a similar time line as the US i.e. approval and revenues in H1 2017 or so.

The orphan drug status however should speed this up- how much, I cannot tell. Best guestimate- 6 to 9 months and the benefit of getting centralized attention within EMA, which is pretty damn good for the whole platform. :woohoo: :vegas:

Best thing though- is that CONDITIONAL APPROVAL (i.e. the hospital exemption etc etc) would apply too and patients could be treated already late 2015 outside of the trial if doctors apply for their patients to be treated with it and ask for reimbursement..... :grin:

That is not impossible given early results... :nice:
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Orphan drug status for Scleroderma in EU 24 Feb 2015 09:23 #3489

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Don't the suffer from Scleroderma in Japan ?

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Orphan drug status for Scleroderma in EU 24 Feb 2015 09:31 #3491

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Don't they suffer from Scleroderma in Japan ?


Sure- although we know its an orphan disease (see my article on it) -orphan drug designation was never talked about by Hedrick & Co.

Obviously they knew too and applied for the designation with a result likely in the works which John/Rongside has written about many times on this board. Just search for scleroderma on the board- the search works very well. :grin:

By the same token- it would be a surprise if nothing was done in Japan with this disorder, which is perfect for ADRCs. :yep:

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Orphan drug status for Scleroderma in EU 25 Feb 2015 08:16 #3513

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Just as an add on.

Scleroderma is GLOBALLY an orphan disease. That means that Cytori most likely will also apply for Orphan drug designation with the FDA in the US.

In 2007 legislation was already passed in Europe AND the US to harmonize the procedure in both markets. Meaning that the same application forms are used-

from Wikipedia:

In an attempt to simplify the process for obtaining orphan status for medications targeting rare diseases, the FDA and the European Medicines Agency (EMA) have created a common application. ... U.S. and European regulators still will conduct independent reviews of application submissions to ensure the data submitted meet the legal and scientific requirements of their respective jurisdictions, the agencies said.


Surely - yesterdays news is coming for the US too. :whistle: :nice:
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Orphan drug status for Scleroderma in EU 25 Feb 2015 09:29 #3514

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Thanks Fas....agree
Also, I believe Hedrick mentioned the possibility about orphan drug pricing...in other words premium pricing.

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Orphan drug status for Scleroderma in EU 25 Feb 2015 09:33 #3515

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Fas, with the price of consumables and machines going down, is it fair to say company will make about 1 K $ per treatment? and maybe 30 thousand patients on a yearly basis, to break even? From the presentations, I think I remember a lot more than that (not total, but new cases per year).

I wonder how Okyanos is doing. I remember hitting one case a day (almost)... would love to see some testimonies from the people. I remember them saying not going to post any, unless we want to contact a few of them personally, with their permission.

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Orphan drug status for Scleroderma in EU 25 Feb 2015 12:38 #3521

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cytxer wrote: Fas, with the price of consumables and machines going down, is it fair to say company will make about 1 K $ per treatment? and maybe 30 thousand patients on a yearly basis, to break even? From the presentations, I think I remember a lot more than that (not total, but new cases per year).

I wonder how Okyanos is doing. I remember hitting one case a day (almost)... would love to see some testimonies from the people. I remember them saying not going to post any, unless we want to contact a few of them personally, with their permission.


Don't forget -- cost going down does not mean price has to go down. The only reason to lower the price is to get more users. In any case, profit must be bigger if the cost of the platform goes down.

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Orphan drug status for Scleroderma in EU 28 Feb 2015 08:14 #3577

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I wonder when we can expect a broadening of the trial beyond scleroderma to encompass rheumatoid arthritis. RA is a huge indication with many more sufferers who generate humongous profits for BP which peddles a wide number of blockbuster products to adress the indication........... inadequately.

www.wisegeek.com/what-is-the-relationship-between-scleroderma-and-rheumatoid-arthritis.htm

Certainly worth following up with a small initial stage II trial to get proof of concept. This would be very substantial and the broader market has no idea. Certainly some of our new big investors are not quite so naive.

www.medpagetoday.com/resource-center/rheumatoid-arthritis/indirectcosts/a/38227

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Orphan drug status for Scleroderma in EU 28 Feb 2015 09:38 #3579

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Learnings from a recent conversation about Scleroderma (not Cytori management): Cytori is focused on getting approval to treat the hand dysfunction associated with Scleroderma. What kills the patients is the growth of collagen inside the chest cavity that crushes vital organs. There may be minor leakage from the injections to the fingers into the blood stream, but a complete treatment for the patient would include intravenous injection(s) to treat the entire body. The "practice of medicine" may result in a "full can of Coke's" worth of adipose tissue being processed to treat the fingers and the whole body. This would not result in additional consumable sales per patient, but it would result in more patients being treated prior to them reaching the point of severe hand dysfunction. Interesting postulation for the long term treatment of autoimmune diseases.

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Orphan drug status for Scleroderma in EU 01 Mar 2015 03:41 #3590

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DOV-

To my knowledge, systemic or diffuse scleroderma is much rarer, since the disorder does not spread that often.

However- in my view- IV treatment of diffuse should be very well possible- also in this day and age with the "right to try" laws in the US, which my friends from diverse activists groups in the Adult Stem Cell communities keep bombarding me with ( I obviously like to be bombarded with that kind of info).

Another factor being VERY favorable for the disorder, is that diffuse scleroderma tends to be the worst around the lungs and thats where most cells first travel to. :whistle:
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