The U.S. Meals and Drug Administration (FDA) has granted Orphan Drug (ODD) and Uncommon Pediatric Illness Designation (RPDD) Standing to Cellectis’ UCART22 product candidate for the remedy of Acute Lymphoblastic Leukemia (ALL).
ALL represents about 10% of all leukemia instances in america, progresses quickly, and is usually deadly inside weeks or months if left untreated. It’s estimated that 6,660 new instances of ALL and 1,560 deaths associated to the illness occurred within the US in 20222.
UCART22 is an allogeneic CAR T-cell product candidate focusing on CD22 and evaluated in BALLI-01, a Section 1/2 open-label dose-escalation and dose-expansion research, designed to guage the security, enlargement, persistence and medical exercise of UCART22 in sufferers with relapse/refractory ALL.
The final medical information introduced by Cellectis on the American Society of Hematology in December 2023 had been encouraging and instructed that UCART22-P2 (absolutely manufactured at Cellectis) is stronger with a preliminary response charge of 67% at Dose Degree 2, in comparison with a 50% response charge at Dose Degree 3 with UCART22-P1 (manufactured by an exterior CDMO). Cellectis expects to offer updates on the progress of BALLI-01 by year-end 2024.
Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis stated: “We’re excited that the FDA granted UCART22 each ODD and RPDD Standing within the remedy of acute lymphoblastic leukemia. This resolution represents further proof of the potential of UCART22 to convey a much-needed therapeutic possibility to those sufferers with ALL. There’s an pressing must develop new therapies for ALL for sufferers who usually are not candidates for HSCT or relapse after CD19 directed CAR T-cell therapies and/or HSCT.”
