Bristol-Myers Squibb Company — Breyanzi EMA EPAR variation assessment report (EMEA/H/C/004731, 2022)
Bristol-Myers Squibb Company · 2022 · EMA EPAR variation assessment report · company profile →
Report summary
The EMA assessed Breyanzi (liso-cel) for pediatric relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) and B-cell non-Hodgkin Lymphoma (B-NHL) in the JCAR017-BCM-004 study. This Phase 1/2 trial was terminated early due to a product-specific waiver. Efficacy in B-ALL (N=11) showed a 45.5% Objective Response Rate (CRi only) with a 13.7-month median Duration of Response. However, safety was concerning, with all 14 subjects experiencing at least one treatment-emergent adverse event, and 85.7% experiencing Grade 3/4 events. The study was halted due to suboptimal response duration, unfavorable adverse events including early cerebral edema, and a narrow therapeutic window, preventing a safe and effective dose. The EMA concluded liso-cel offered no significant therapeutic benefit over existing treatments like tisa-cel in this pediatric population.
Key findings — cited to the page
| subjects experiencing at least 1 NCI CTCAE Grade 3/4 TEAE | 85.7 % | BCM-004 · p.18 |
| subjects experiencing at least 1 NCI CTCAE Grade 3/4 TEAE related to liso-cel | 57.1 % | BCM-004 · p.18 |
| Grade 3/4 Treatment-Emergent Adverse Events (TEAE) | 12/14 subjects | BCM-004 · p.27 |
| median Duration of Response (DOR) | 13.7 months | BCM-004 · p.14 |
| planned enrolment | 101 subjects | BCM-004 · p.9 |
| Objective Response Rate (ORR) | 45.5 % | BCM-004 · p.14 |
| study phase | Phase 1/2 | JCAR017-BCM-004 · p.4 |
| subjects experiencing at least 1 TEAE | 100 % | BCM-004 · p.18 |
| median Overall survival (OS) | 8.90 months | BCM-004 · p.16 |
| initial starting dose of liso-cel | 0.50 x 10^6 CAR+ T cells/kg | BCM-004 · p.7 |
| amended starting dose of liso-cel | 0.05 x 10^6 CAR+ T cells/kg | BCM-004 · p.7 |
| MRD-negative response rate | 27.3 % | BCM-004 · p.14 |
Source: Bristol-Myers Squibb Company ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Oncology library →