Quark Pharmaceuticals, Inc., has announced that the European Commission has granted Orphan Medicinal Product Designation for QPI-1002 (also referred to as “I5NP”) for the prophylaxis of delayed graft function (DGF) in kidney transplant patients. QPI-1002 is a synthetic siRNA targeting p53 mRNA and is the first synthetic siRNA to be administered systemically to humans. The designation is granted under the name of the European sponsor, Verius, Ltd. of the United Kingdom.
Dr. Daniel Zurr, Quark’s Chief Executive Officer, stated, “The addition of orphan designation for the European Union, following the US FDA orphan designation for Quark’s QPI-1002 announced in February, provides the opportunity for regulatory support for this indication in that region as well. We believe the EU renal transplant community will welcome development of QPI-1002 with the same enthusiasm already demonstrated in the US. A therapy that succeeds in reducing the incidence and severity of DGF may improve longer-term graft survival and help to reduce the widening gap between donor organ availability and the number of patients awaiting transplantation.” Orphan designation in the EU grants special status to a product to treat a rare disease or condition affecting less than five in 10,000 persons in the EU per year. The provisions for the EU orphan designation allow for incentives to pharmaceutical companies developing such products, thus providing better access to treatments for patients having conditions that otherwise might not be investigated. For marketing approved products this includes potential market exclusivity of up to 10 years in the EU as well as various fee reductions for certain regulatory activities.
