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Monday, March 31, 2014

FDA Grants Orphan Drug Status to Anti-CD19 T Cell Therapy for DLBCL


The U.S. Food and Drug Administration, today, has granted orphan drug designation to an investigational autologous engineered T cell therapy, anti-CD19 chimeric antigen receptors (CAR), for the treatment of diffuse large B cell lymphoma (DLBCL). The product genetically modifies patients’ own white blood cells to recognize and target CD19 expression on B cell malignancies.


Early clinical trials data were reported at the American Society of Hematology Annual Meeting in December (Abstract 168), and further phase I and II clinical trials for the therapy are ongoing.


The Orphan Drug designation—to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.—grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.


The treatment is manufactured by Kite Pharma, Inc., and is being developed through a cooperative research and development agreement (CRADA) in partnership with the National Cancer Institute Surgery Branch.