Cellectis’ UCART22 Granted ODD and RPDD Status by FDA

Cellectis’ UCART22 Granted ODD and RPDD Status by FDA

By, Admin 29 July 2024

Overview

UCART22 is an allogenic CAR T-cell product candidate for the treatment of Acute Lymphoblastic Leukemia. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug (ODD) and Rare Pediatric Disease Designation (RPDD) Status to Cellectis’ UCART22 product candidate for the treatment of Acute Lymphoblastic Leukemia (ALL).

Leukemia in US

  • ALL represents about 10% of all leukemia cases in the United States, progresses rapidly, and is typically fatal within weeks or months if left untreated. 
  • It is estimated that 6,660 new cases of ALL and 1,560 deaths related to the disease occurred in the US in 20222.

About UCART22

UCART22 is an allogeneic CAR T-cell product candidate targeting CD22 and evaluated in BALLI-01, a Phase 1/2 open-label dose-escalation and dose-expansion study, designed to evaluate the safety, expansion, persistence and clinical activity of UCART22 in patients with relapse/refractory ALL.

Cellectis Latest Clinical Data

  • The last clinical data presented by Cellectis at the American Society of Hematology in December 2023 were encouraging and suggested that UCART22-P2 (fully manufactured at Cellectis) is more potent with a preliminary response rate of 67% at Dose Level 2, compared to a 50% response rate at Dose Level 3 with UCART22-P1 (manufactured by an external CDMO). 
  • Cellectis expects to provide updates on the progress of BALLI-01 by year-end 2024.

Words from CMO: Cellectis

Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis said: “We are excited that the FDA granted UCART22 both ODD and RPDD Status in the treatment of acute lymphoblastic leukemia. This decision represents additional evidence of the potential of UCART22 to bring a much-needed therapeutic option to these patients with ALL. There is an urgent need to develop new therapies for ALL for patients who are not candidates for HSCT or relapse after CD19 directed CAR T-cell therapies and/or HSCT.”

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