Juno Therapeutics, Inc. announced today that the FDA has granted Orphan Drug Designation to its JCAR015 chimeric antigen receptor product candidate. The designation was granted for treatment of acute lymphoblastic leukemia. JCAR015 Phase I trials are currently underway at Juno's collaboration partner, Memorial Sloane Kettering Cancer Center.
Orphan drug designations are granted to drugs intended to treat rare diseases or conditions, or "orphan" diseases. The designation allows for incentives to advance development and for marketing of drugs that progress to approval.
All three of Juno's CAR T cell product candidates currently in trial, including JCAR015, are based on chimeric antigen receptor technology that employs the body's immune system to attack cancer cells. JCAR017, in Phase I/II trials at Seattle Children's Hospital, is being tested for pediatric and young adult relapsed/refractory CD19 positive leukemia. JCAR014, currently in Phase I/II trials at the Fred Hutchison Cancer Research Center, is being tested for relapsed or refractory chronic lymphocytic leukemia, non-Hodgkin's lymphoma, and acute lymphoblastic leukemia.
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