FDA Grants Orphan Drug Designation for Anti-CD19 T-Cell Cancer Immunotherapy Product

“Kite Pharma, Inc, announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development 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.

“Orphan drug designation is granted by the FDA Office of Orphan Products Development 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 United States. The orphan drug designation also would entitle Kite Pharma to a 7-year period of marketing exclusivity in the United States.”

Editor’s note: When a newly developed drug for a rare (“orphan”) disease seems particularly promising for patients, the FDA may choose to grant it “orphan drug designation.” The designation removes certain barriers that might otherwise keep a drug company from being able to successfully develop and profit from an orphan drug. A new treatment for diffuse large B-cell lymphoma has now been granted orphan drug designation.