Kite previously announced in March 2014 that the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation for KTE-C19 for the treatment of DLBCL. Further, Kite has filed an investigational new drug (IND) application to conduct a Phase 1/2 clinical trial for KTE-C19 with the FDA.
"We are pleased with the approval of orphan drug designation for KTE-C19 in the EU, another important milestone for Kite Pharma and for the progress of our lead program," said Arie Belldegrun, M.D., President and Chief Executive Officer of Kite Pharma.
"DLBCL is an aggressive blood cancer that represents a significant unmet need for those patients with refractory or relapsed disease. The KTE-C19 program is part of Kite’s broad commitment to delivering innovative therapies that potentially can transform the lives of cancer patients around the world."
Orphan drug designation by the European Commission provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and where no satisfactory treatment is available.
In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to the centralized authorization procedure.
KTE-C19 is Kite Pharma’s lead product candidate in which a patient’s T cells are genetically modified using a gammaretroviral vector to express a chimeric antigen receptor (CAR) designed to target the antigen CD19, a protein expressed on the cell surface of B cell lymphomas and leukemias.