FDA approves Breyanzi CAR-T for chronic lymphocytic leukemia, small lymphocytic lymphoma
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The FDA granted accelerated approval to lisocabtagene maraleucel for treatment of certain patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma.
The indication applies to adults who received at least two prior lines of therapy, including a Bruton tyrosine kinase inhibitor (BTKi) and a B-cell lymphoma 2 inhibitor (BCL2i).
The FDA granted accelerated approval to lisocabtagene maraleucel for treatment of certain patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma. Image: Adobe Stock
Lisocabtagene maraleucel (Breyanzi, Bristol Myers Squibb) — often called liso-cel — is a CD19-directed chimeric antigen receptor T-cell therapy.
The FDA previously approved liso-cel for treatment of adults with relapsed or refractory large B-cell lymphoma who received at least one prior therapy.
There is no standard of care for patients with relapsed or refractory CLL or small lymphocytic lymphoma (SLL) previously treated with targeted agents.
The phase 1/phase 2 TRANSCEND CLL 004 trial evaluated liso-cel for patients with relapsed or refractory CLL or SLL. Eighty-nine patients received the recommended phase 2 dose of 100 x 106 CAR T cells.
Complete response rate — including complete remission with incomplete bone marrow recovery — served as the primary endpoint.
Researchers reported a 45% (95% CI, 32.3-57.5) overall response rate and a 20% (95% CI, 11.1-31.8) complete response rate. Median duration of response was 35.3 months (95% CI, 12.4 to not reached) among all patients who achieved response but had not been reached among those who achieved complete response (95% CI, 15 months to not reached).
Among those who achieved complete response, results showed a 100% (95% CI, 75.3-100) minimal residual disease (MRD) negativity rate in the blood and a 92.3% (95% CI, 64-99.8) MRD negativity rate in the bone marrow.
Seventy-four (83%) study participants developed cytokine release syndrome; however, most were low grade, with 9% of cases classified as grade 3 and no cases classified as grade 4/grade 5.
Slightly less than half (46%) of study participants experienced neurologic events, with 20% classified as grade 3. Researchers reported one grade 4 neurologic event and no grade 5 events.
“CLL and SLL are currently considered incurable diseases with few treatment options in the relapsed setting that can confer complete responses, something that has historically been associated with improved long-term outcomes,” Tanya Siddiqi, MD, lead investigator of TRANSCEND CLL 004 and associate professor in the division of lymphoma at City of Hope, said in a Bristol-Myers Squibb press release. “The FDA approval of liso-cel in relapsed or refractory CLL and SLL after treatment with prior BTKi and BCL2i is a remarkable breakthrough, shifting the treatment paradigm from continuous therapy with sequential regimens to overcome drug resistance, to a one-time personalized T-cell based approach that has the potential to offer patients complete and lasting remission.”
Continued approval for this indication may be contingent on continued demonstration of clinical benefit in a confirmatory trial.
Liso-cel includes boxed warnings about the potential for cytokine release syndrome, neurologic toxicities and secondary hematologic malignancies.
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