Breyanzi Receives Positive CHMP Opinion for Follicular Lymphoma Treatment in EU
核心洞察
The CHMP has recommended Breyanzi for treating relapsed or refractory follicular lymphoma (搜索) (FL) in adults after two or more prior systemic therapies.
The recommendation is based on the Phase 2 TRANSCEND FL study, which showed a 97.1% overall response rate and a 94.2% complete response rate.
Breyanzi demonstrated rapid and durable responses, with 75.7% of patients in response at 18 months, and a consistent safety profile in clinical trials.
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the approval of Breyanzi (lisocabtagene maraleucel; liso-cel), a CD19 (搜索)-directed CAR T cell therapy, for treating adult patients with relapsed or refractory follicular lymphoma (搜索) (FL) after two or more prior lines of systemic therapy. This decision marks a significant step toward expanding treatment options for this incurable form of non-Hodgkin lymphoma (搜索) (NHL). The European Commission (EC) will now review the CHMP recommendation, with a final decision expected within approximately two months.
TRANSCEND FL Study Results
The positive opinion is based on data from the global, Phase 2 TRANSCEND FL study (NCT04245839), which is the largest clinical trial to date evaluating a CAR T cell therapy in patients with relapsed or refractory indolent NHL, including FL. The study enrolled adults with relapsed or refractory FL who had received two or more prior lines of systemic therapy.
Breyanzi demonstrated a high overall response rate (ORR) of 97.1% (95% CI: 91.7–99.4) and a complete response rate (CR) of 94.2% (95% CI: 87.8–97.8), meeting the study’s primary and key secondary endpoints, respectively. Responses were rapid and durable, with 75.7% (95% CI: 66.0–83.0) of patients in response at 18 months. The safety profile of Breyanzi in FL was consistent with its well-established safety profile observed across clinical trials, with no new safety signals identified.
Follicular Lymphoma: An Unmet Need
Follicular lymphoma (搜索) is the second most common form of NHL, accounting for 20-30% of all NHL cases. It is characterized by cycles of remission and relapse, and while advancements have been made in FL treatment, relapsed or refractory FL remains an area of high unmet need. Approximately 20% of patients experience disease relapse or progression within two years of first-line treatment. CAR T cell therapies offer the potential for lasting results in routine care by providing high rates of complete, durable responses and a manageable safety profile.
Breyanzi: A CAR T Cell Therapy
Breyanzi is a CD19 (搜索)-directed CAR T cell therapy with a 4-1BB costimulatory domain, enhancing the expansion and persistence of CAR T cells. It is made from a patient’s own T cells, which are collected and genetically re-engineered to target CD19-expressing cells. The modified T cells are then infused back into the patient as a one-time treatment.
Breyanzi is already approved in the EU for treating adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL (搜索)), high grade B-cell lymphoma (HGBCL (搜索)), primary mediastinal large B-cell lymphoma (PMBCL (搜索)), and FL grade 3B (FL3B) under specific conditions. The CHMP recommendation could broaden its application to include a wider range of FL patients.
Safety and Efficacy Considerations
While Breyanzi has demonstrated promising efficacy, it is associated with potential risks, including Cytokine Release Syndrome (CRS) and neurologic toxicities. These risks are managed through a Risk Evaluation and Mitigation Strategy (REMS) program. Clinical trials have reported varying rates of CRS and neurologic toxicities, emphasizing the need for careful monitoring and management.
Future Implications
If approved by the EC, Breyanzi could offer a valuable new treatment option for patients with relapsed or refractory FL in the EU, addressing a significant unmet medical need. Bristol Myers Squibb (搜索) continues to explore Breyanzi's potential in other types of lymphoma through ongoing clinical studies.