The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for lisocabtagene maraleucel (liso-cel; Bristol-Myers Squibb) for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after at least 2 prior therapies.

Liso-cel is an autologous anti-CD19 chimeric antigen receptor (CAR) T-cell immunotherapy with a defined composition of CD8+ and CD4+ CAR T cells. The BLA is supported by data from the pivotal phase 1 study (TRANSCEND NHL 001) that assessed the safety, antitumor activity, and pharmacokinetics of liso-cel in 268 patients with relapsed or refractory large B-cell non-Hodgkin lymphoma (NHL), including diffuse large B-cell lymphoma (DLBCL), high-grade lymphoma (HGL), primary mediastinal B-cell lymphoma (PMBCL), and Grade 3B follicular lymphoma (FL). 

The primary end point included treatment-related adverse events, dose-limiting toxicities and objective response rate. Key secondary end points included complete response rate, duration of response (DOR), and progression-free survival (PFS). 

Findings from the study demonstrated an overall response rate of 73% (n=187 of 256; 95% CI, 67-78), including 53% of patients (n=136 of 256; 95% CI, 47-59) achieving a complete response (CR). The median DOR was not reached for all patients (95% CI, 8.6 months-NR) at a median follow-up of 12 months (95% CI, 11.2-16.7). Moreover, median PFS and median overall survival were 6.8 months (95% CI, 3.3-14.1) and 21.1 months (95% CI, 13.3-NR), respectively. Treatment-emergent adverse events of grade 3 or higher were observed in 79% of patients; these included neutropenia, anemia, and thrombocytopenia.

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“Based on the TRANSCEND NHL 001 data, liso-cel has the potential to expand treatment options for those affected by this aggressive blood cancer who did not respond to initial therapies or whose disease has relapsed,” said Stanley Frankel, MD, senior VP, Cellular Therapy Development, Bristol-Myers Squibb. “This BLA acceptance and Priority Review designation is an important step as we work to improve treatment for these patients in need.”

A Prescription Drug User Fee Act (PDUFA) target date of August 17, 2020 has been set for this application.

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