Cytori Receives Positive European Opinion on Scleroderma Orphan Drug Status
Cytori Therapeutics, Inc. recently announced that the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) has given a positive opinion concerning Cytori’s submission for an orphan drug designation for ECCS-50, the company’s experimental Scleroderma therapeutic, which is based on stromal vascular fraction cells, from adipose tissue and derived from Celution® System, Cytori’s proprietary technology.
Cytori recently received the FDA’s approval to evaluate ECCS-50 for Scleroderma hand dysfunction in an eighty-patient Phase 3 pivotal study. The trial will be enrolling patients in 12 different locations across the United States this year.
Marc Hedrick, CEO and president of Cytori Therapeutics, said in a press release: “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. 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. 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,” said Ken Kleinhenz from Cytori.
Companies can file a single application with the European Medicines Agency in order to have an orphan medicine assessed in order for the European Commission to make a decision that would be valid in all EU Member States. A conditional approval is also available to orphan medicines. Once they are approved, orphan medicines have a decade-long protection from any market competition with similar medicines with similar indications.
Professor Brigitte Granel, first author of the publication on the outcomes of the SCLERADEC–I trial said in a press release: “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.”