Kite Pharma, Inc., a clinical-stage biopharmaceutical company, announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has adopted positive opinions recommending KTE-C19 for designation as an orphan medicinal product for the treatment of acute lymphocytic leukemia (ALL), chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) and follicular lymphoma (FL).
KTE-C19 is an investigational therapy in which a patient's T cells are genetically modified 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. Kite previously received orphan drug designation for KTE-C19 for the treatment of diffuse large B-cell lymphoma (DLBCL) in both the US and the EU, as well as COMP positive opinions for orphan drug designation in the EU for primary mediastinal B-cell lymphoma (PMBCL) and mantle cell lymphoma (MCL).
"The orphan drug designation is an important regulatory milestone as we further our development of KTE-C19 in advanced haematological cancers, where there are limited treatment options," said Arie Belldegrun, M.D., FACS, chairman, president, and chief executive o.
"We are excited by the progress of our ongoing phase 1/2 clinical trial (ZUMA-1) of KTE-C19 in patients with refractory, aggressive non-Hodgkin lymphoma, including DLBCL, PMBCL and transformed follicular lymphoma (TFL), for which we anticipate presenting top-line phase 1 data later this year."
The COMP adopts an opinion on the granting of orphan drug designation, after which the opinion is submitted to the European Commission (EC) for endorsement of the opinion. Orphan drug designation by the EC 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 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 authorisation procedure.