FDA Gives Priority Review to BMS’s Supplemental BLA for Liso-Cel in R/R Marginal Zone Lymphoma

Bristol Myers Squibb’s supplemental biologics license application (sBLA) for marketed autologous CD19-directed CAR T-cell therapy lisocabtagene maraleucel (liso-cel, marketed as Breyanzi) for marginal zone lymphoma (MZL) has been accepted by the FDA with priority review.¹ The Prescription Drug User Fee Act (PDUFA) goal date for the sBLA has been set for December 5, 2025.

Specifically, the sBLA is under review for adults with relapsed/refractory (r/r) MZL who have been treated with 2 or more previous lines of systemic therapy. The sBLA is supported by data from thephase 2 TRANSCEND FL clinical trial (NCT04245839).

“While initial therapy for MZL can be effective, multiple relapses over the course of several years are common, leaving patients in need of a new treatment option that can provide high, lasting response rates,” Rosanna Ricafort, the vice president and head of Late Development Program Leadership, Hematology and Cell Therapy, at BMS, said in a statement.¹ “This FDA acceptance brings us one step closer to potentially standardizing CAR T-cell therapy as a treatment option for MZL, while building on our commitment to bring this personalized therapy to as many eligible patients as possible.”

Results from TRANSCEND FL’s MZL cohort were recently presented at the 2025 International Conference on Malignant Lymphoma (ICML), held June 17 to June 21 in Lugano, Switzerland.² Among the 66 efficacy evaluable patients with r/r MZL who were treated with liso-cel, the overall response rate (ORR) was 95.5% (95% CI: 87.3-99.1; one-sided P <.0001) and the complete response rate was 62.1% (95% CI: 49.3-73.8; one-sided P < .0001). The 24-month rate for duration of response was 88.6% (median follow-up, 21.6 months), the 24-month rate for progression-free survival was 85.7% (median follow-up, 23.8 months), and the 24-month rate for overall survival was 90.4% (median follow-up, 24.5 months).

“Liso-cel achieved high, lasting response rates in patients with r/r MZL, underscoring the potential of this one-time therapy to significantly improve patient outcomes,” TRANSCEND FL study investigator M. Lia Palomba, MD, a lymphoma and cell therapy specialist at Memorial Sloan Kettering Cancer Center, said in a June 2025 statement.² “Currently, the median survival for patients with marginal zone lymphoma with multiple relapses is 3 to 5 years, signifying an urgent need for transformative therapies that can effectively address this hard-to-treat disease.”

With regard to safety, cytokine release syndrome (CRS) was reported in 76% of patients, with 4% of patients experiencing grade 3 CRS cases. There were no cases of grade 4 or 5 CRS reported. Neurologic events occurred in 33% of patients, with 4% of patients experiencing grade 3 NE cases. There were no grade 4 or 5 NE cases reported. ORR and safety analyses were ongoing at the time the data were reported.

“MZL is an indolent disease but remains an area of high unmet need for patients who are relapsing and reaching later lines of treatment,” Ricafort added to Palomba’s statement.² “We are proud to present for the first time the primary analysis data from the MZL cohort of TRANSCEND FL, underscoring our commitment to unlock the full potential of cell therapy to help patients living with r/r lymphomas. As highlighted by the data at ICML, Breyanzi continues to cover the broadest patient eligibility of any CAR-T for B-cell malignancies and demonstrates a safety profile consistent with clinical trials and in the real-world setting for approved indications.”

REFERENCES
1. Bristol Myers Squibb’s application for Breyanzi (lisocabtagene maraleucel) accepted for priority review by U.S. Food and drug administration (FDA) in fifth cancer type for relapsed or refractory marginal zone lymphoma (MZL). News release. Bristol Myers Squibb. August 4, 2025. Accessed August 4, 2025. https://news.bms.com/news/corporate-financial/2025/Bristol-Myers-Squibbs-Application-for-Breyanzi-lisocabtagene-maraleucel-Accepted-for-Priority-Review-by-U-S--Food-and-Drug-Administration-FDA-in-Fifth-Cancer-Type-for-Relapsed-or-Refractory-Marginal-Zone-Lymphoma-MZL/default.aspx
2. Bristol Myers Squibb presents first data from the marginal zone lymphoma cohort of the TRANSCEND FL trial demonstrating deep and durable responses with Breyanzi (lisocabtagene maraleucel). News release. Bristol Myers Squibb. June 16, 2025. Accessed August 4, 2025. https://news.bms.com/news/corporate-financial/2025/Bristol-Myers-Squibb-Presents-First-Data-from-the-Marginal-Zone-Lymphoma-Cohort-of-the-Transcend-FL-Trial-Demonstrating-Deep-and-Durable-Responses-with-Breyanzi-lisocabtagene-maraleucel/default.aspx