Cytori Therapeutics Inc (NASDAQ:CYTX) announced 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 deMarseille 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 Commission5:
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 (Original Source)
Shares of Cytori Therapeutics closed yesterday at $1.32 . CYTX has a 1-year high of $2.63 and a 1-year low of $0.36. The stock's 50-day moving average is $1.08 and it's 200-day moving average is $0.65.
On the ratings front, Cytori Therapeutics has been the subject of a number of recent research reports. In a report released yesterday, Laidlaw analyst Yale Jen reiterated a Buy rating on CYTX, with a price target of $4, which represents a potential upside of 203.0% from where the stock is currently trading. Separately, on the same day, Roth Capital's Joseph Pantginis maintained a Buy rating on the stock and has a price target of $6.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Yale Jen and Joseph Pantginis have a total average return of 52.0% and 12.1% respectively. Jen has a success rate of 75.0% and is ranked #501 out of 3568 analysts, while Pantginis has a success rate of 53.6% and is ranked #126.
Cytori Therapeutics Inc is engaged in the development of novel treatments for cardiovascular disease and soft tissue injuries and burns.