FDA Accepts Priority Review Application for Lisocabtagene Maraleucel for Relapsed or Refractory CLL, SLL


The acceptance comes after results from TRANSCEND CLL 004, which is the first trial to demonstrate clinical benefit with a CAR T cell therapy in patients who were previously treated for CLL or SLL.

The FDA has accepted the supplemental Biologics License Application (sBLA) for lisocabtagene maraleucel (Breyanzi; Bristol-Myers Squibb), expanding its current indication to include the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who received a prior Bruton tyrosine kinase inhibitor (BTKi) and B-cell lymphoma 2 inhibitor (BCL2i). Further, the FDA has granted the application Priority Review, underscoring the high unmet need and the significant advancement lisocabtagene maraleucel could offer this patient population which lacks standard of care and treatment options.

3D illustration of leukemia

Image credit: Dr_Microbe | stock.adobe.com

Lisocabtagene maraleucel is approved by the FDA for the treatment of adult patients with large B-cell lymphoma. It is a CD19-directed CAR T cell therapy with a 4-1BB costimulatory domain that enhances the expansion and persistence of the CAR T cells. Lisocabtagene maraleucel is created by collecting and re-engineering the patient’s T cells to become CAR T cells, which are delivered through infusion as a 1-time treatment.

“Currently, there is no standard of care for people living with relapsed or refractory CLL or SLL after treatment with BTKi- and BCL2i-based regimens, leaving a critical unmet need for a treatment option that provides deep and lasting responses,” said Anne Kerber, senior vice president and head of Late Clinical Development, Hematology, Oncology, Cell Therapy (HOCT) at Bristol Myers Squibb, in a press release.

The application came after results from the TRANSCEND CLL 004 study (NCT03331198), a pivotal open-label, single-arm, multicenter phase 1/2 study. The study was the first to demonstrate clinical benefit with a CD19-directed CAR T cell therapy in patients with relapsed or refractory CLL or SLL after progression following treatment with a BTKi and BCL2i.

The phase 1 dose escalation portion of the study evaluated the safety and recommended dose for the subsequent phase 2 expansion cohort. The phase 2 portion evaluated lisocabtagene maraleucel at the recommended dose from the phase 1 monotherapy arm. The primary endpoint of the phase 2 portion of the study was complete response rate—including complete remission with incomplete bone marrow recovery—based on independent review committee according to International Workshop on CLL guidelines.

The most common nonlaboratory adverse events (AEs) reported by patients who received treatment with lisocabtagene maraleucel were fever, cytokine release syndrome, fatigue, musculoskeletal pain, and nausea. Further, the most common grade 3 and 4 AEs included lymphocyte count disease, neutrophil count decrease, platelet count decrease, and hemoglobulin decrease. Other reported AEs included neurologic toxicities, hypersensitivity reactions, infections, prolonged cytopenias, and hypogammaglobulinemia.

“This FDA acceptance brings us 1 step closer to offering these patients, for the first time, a personalized, T-cell based treatment option. We’re proud to further our commitment to bring the potential of CAR T cell therapy to more patients, building on [lisocabtagene maraleucel] foundation as a differentiated treatment option that has shown clinical benefit in the broadest array of B-cell malignancies,” said Kerber in the press release.


Bristol-Myers Squibb. U.S. Food and Drug Administration Accepts for Priority Review Bristol Myers Squibb’s Application for Breyanzi (lisocabtagene maraleucel) for Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL). News release. November 9, 2023. Accessed November 14, 2023. https://www.businesswire.com/news/home/20231109738749/en

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