Lisocabtagene maraleucel (Breyanzi), a CAR T cell therapy for B-cell lymphomas, has been approved by the US FDA to treat adults with marginal zone lymphoma (MZL). The treatment, made by BMS subsidiary Juno Therapeutics was previously granted Priority Review and Orphan Drug designations for its application.

A week of positive news

The news comes in the same week as positive results for the therapy’s efficacy against follicular lymphoma in a three year follow-up from the TRANSCEND FL trial. In that trial, a significant number of patients with relapsed or refractory follicular lymphoma achieved durable multi-year remission after exhausting other treatment options.

Sairah Ahmed, an Associate Professor at MD Anderson and principal investigator on the trial said: “These three-year results highlight not only the long-lasting benefit of CAR T cell therapy, but also its favourable safety profile, offering real hope for patients facing this challenging disease. MD Anderson is a participating site of the TRANSCEND trial.”

Marginal zone lymphoma

Lisocabtagene maraleucel’s new approval concerns its use in adults with marginal zone lymphoma in adults who have failed treatment with or relapsed after two or more prior lines of therapy. It is the first CAR T cell therapy to be approved for the indication.

MZL is a rare and slow growing lymphatic cancer with poor average survival outcomes in relapsed or refractory cohorts. Approximately 7% of all B-cell non-Hodgkin lymphomas in the US are MZL, about 7,460 year-on-year.

“In granting approval, FDA took into consideration the high and durable response rate following a one-time treatment in patients with MZL, almost half of whom had either progression within two years of diagnosis or had refractory disease,” said Vijay Kumar, an Acting Director at FDA’s Center for Biologics Evaluation.

Lynelle B. Hoch, president, Cell Therapy Organization, Bristol Myers Squibb said: “Breyanzi is the first and only CAR T cell therapy approved for this patient population, demonstrating Bristol Myers Squibb’s deep commitment to expanding access and reaching as many patients as possible with this innovative, practice-changing treatment.”

TRANSCEND FL

This approval is based on results from the TRANSCEND FL, an open-label, multicentre, multi-cohort, single-arm study. Study participants underwent leukapheresis to collect a patient’s own immune cells which were then used to manufacture Breyanzi. After patients finished preparatory chemotherapy to reduce their lymphocyte levels, they received a single dose of Breyanzi within two to seven days.

Among the 77 patients who underwent leukapheresis, 66 patients received the study specified single infusion of Breyanzi, of which 95.5% of patients experienced a response to treatment and 62.1% of patients had complete response without signs of marginal zone lymphoma on imaging scans. The responses were durable after a median follow up of 21.6 months.