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Liso-cel was approved in the EU for relapsed/refractory MCL after at least 2 lines of therapy, including a BTK inhibitor.
The European Commission has approved lisocabtagene maraleucel (Breyanzi; liso-cel)for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) following at least 2 prior lines of systemic therapy, including a BTK inhibitor.1
This regulatory decision is supported by data from the MCL cohort of the phase 1 TRANSCEND NHL 001 trial (NCT02631044), which evaluated the CD19-directed CAR T-cell therapy in a heavily pretreated population with high unmet need.
In this cohort, liso-cel demonstrated robust activity, yielding an overall response rate (ORR) of 82.7% (95% CI, 72.7%-90.2%) and a complete response (CR) rate of 71.6% (95% CI, 60.5%-81.1%). Responses occurred rapidly, with a median time to first response of 0.95 months, and 41.2% (95% CI, 29.2%-52.9%) of patients remained in response at 24 months.
“This approval for [liso-cel] in relapsed or refractory MCL marks another important step as we continue to deliver on the promise of cell therapy for more eligible patients across Europe [with] the fourth approval for [liso-cel] in Europe,” Emma Charles, senior vice president, Europe Region, Bristol Myers Squibb, stated in a news release. “While frontline therapies have advanced over the years for this rare but aggressive form of non-Hodgkin lymphoma, the vast majority of patients relapse or become resistant and face reduced survival outlook, leaving a critical need for new treatment options. [Liso-cel] has the opportunity to address a treatment gap for this patient population based on its demonstrated clinical benefit.”
In May 2024, the FDA approved liso-cel for the treatment of adult patients with relapsed or refractory MCL who have received at least 2 prior lines of systemic therapy, including a BTK inhibitor.2 This decision was also supported by data from the MCL cohort of TRANSCEND NHL 001.
TRANSCEND NHL 001 was an open-label, multicenter, pivotal phase 1 study employing a single-arm, seamless design to evaluate the safety, pharmacokinetics, and antitumor activity of liso-cel in adult patients with relapsed or refractory B-cell non-Hodgkin lymphoma.3 Within the MCL cohort, patients needed to have a confirmed diagnosis via cyclin D1 expression or evidence of t(11;14) by cytogenetics, fluorescent in situ hybridization [FISH], or PCR. Relapsed/refractory disease after at least 2 prior lines of systemic MCL therapy was required, and prior therapies needed to include an alkylating agent, a BTK inhibitor, and rituximab (Rituxan) or another CD20-targeted agent.
Other key inclusion criteria for all patients included PET-positive disease per Lugano classification, an ECOG performance status of 0 or 1, and adequate bone marrow, renal, hepatic, pulmonary, and cardiac function.
The study evaluated liso-cel on both 1-dose and 2-dose schedules. Primary end points focused on treatment-related adverse effects, dose-limiting toxicities, and ORR. Secondary end points include CR rate, duration of response, progression-free survival, overall survival, and health-related quality of life.
Safety findings from the MCL cohort of TRANSCEND NHL 001 demonstrated that liso-cel maintained the predictable and well-characterized safety profile observed across prior clinical trials and approved indications.1 Toxicities were generally early in onset and resolved with standard management, supporting recent updates that allow for shorter post-infusion monitoring requirements.
Any-grade cytokine release syndrome (CRS) occurred in 61% of patients treated with liso-cel, although grade 3 or 4 CRS was infrequent (1%). The median time to CRS onset was 4 days (range, 1-10). Any neurologic toxicities of any grade were reported in 31% of patients, with grade 3 or 4 effects occurring in 9%. Median time to onset of neurologic effects was 8 days (range, 1-25).
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