Kite Pharma, a clinical-stage biotechnology company focused on developing engineered autologous T cell therapy (eACT) products for cancer, has announced that the FDA Office of Orphan Products Development (OOPD) granted Orphan Drug Designation for the company's lead investigational therapy, an autologous engineered T cell product that targets CD19 expression on B cell malignancies, for the treatment of diffuse large B cell lymphoma (DLBCL).
Orphan Drug Designation is granted by the FDA OOPD to novel drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. The OOPD provides incentives for sponsors to develop products for rare diseases, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. The Orphan Drug Designation also would entitle Kite Pharma to a seven-year period of marketing exclusivity in the U.S. pending FDA approval of Kite's lead product for the treatment of DLBCL.
DLBCL is an aggressive type of non-Hodgkin lymphoma for which the primary treatment options are front-line chemotherapy and, in selected patients, autologous transplant. Although most patients with DLBCL can be cured by either chemotherapy or transplant, a significant proportion of patients have disease that is resistant to chemotherapy. For these patients with relapsed/refractory DLBCL, there is a substantial unmet need for more effective therapies. There are approximately 22,000 new diagnoses of DLBCL in the U.S. each year.