The European Medicines Agency’s Committee for Medicinal Products for Human Use has recommended the approval of lisocabtagene maraleucel for the treatment of select patients with large B-cell lymphoma who relapsed within 12 months from completion of or developed refractory disease to frontline chemoimmunotherapy.
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of lisocabtagene maraleucel (liso-cel; Breyanzi) for the treatment of patients with diffuse large B-cell lymphoma (DLBCL), high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B, who relapsed within 12 months from completion of or developed refractory disease to frontline chemoimmunotherapy.1
The recommendation is based on data from the pivotal phase 3 TRANSFORM trial (NCT03575351), where liso-cel improved event-free survival (EFS), progression-free survival (PFS), and response rates vs standard, platinum-based salvage chemotherapy and high-dose chemotherapy plus hematopoietic stem cell transplant (HSCT) as second-line treatment in patients with relapsed or refractory LBCL.
The European Commission will announce its final decision within approximately two months following receipt of the CHMP opinion. The decision will be applicable to all European Union member states and Iceland, Norway, and Liechtenstein.
“This positive CHMP opinion is an important milestone towards introducing a potential new standard of care for people in the European Union living with relapsed or refractory LBCL after first-line treatment, an area of critical unmet need where few patients are able to undergo or derive long-term clinical benefit from HSCT,” Anne Kerber, senior vice president, Cell Therapy Development, Bristol Myers Squibb, said in a news release. “We look forward to continuing to work with the European Medicines Agency with the goal of bringing our cell therapy, [liso-cel], and the potential for cure to more people.”
On June 24, 2022, the FDA approved liso-cel as second-line therapy for patients with relapsed or refractory LBCL based in part on findings from the TRANSFORM trial.2
To be eligible for enrollment in the pivotal phase 3 study, patients had to have LBCL that was primary refractory or relapsed within 12 months after first-line therapy containing a CD20-antibody and anthracycline.
Patients were randomly assigned to receive liso-cel or rituximab (Rituxan) plus dexamethasone, high-dose cytarabine, and cisplatin (R-DHAP), rituximab plus ifosfamide, carboplatin and etoposide (R-ICE), or rituximab plus gemcitabine, dexamethasone and cisplatin (R-GDP) per the investigator’s choice prior to undergoing high-dose chemotherapy and HSCT.
The primary end point of the study was EFS, defined as time from randomization to death from any cause, progressive disease, failure to achieve complete response or partial response, or start of new antineoplastic therapy due to efficacy concerns. Complete response rate was a key secondary end point. Other efficacy endpoints included PFS, overall survival, overall response rate, and duration of response.
Updated findings from the trial, which were presented at the 2022 ASH Annual Meeting and Exposition with median follow-up of 17.5 months, showed sustained improvements in EFS, PFS, and response rates with liso-cel vs standard therapy.3