The European Commission (EC) has approved an expanded indication for blinatumomab to include the treatment of pediatric patients with Philadelphia chromosome–negative (Ph-negative) relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). The bispecific T-cell engager had previously received approval for treatment of adults with Ph-negative relapsed or refractory B-cell precursor ALL in 2015.
Blinatumomab was granted breakthrough therapy and priority review designations by the U.S. Food and Drug Administration (FDA) in 2014. The FDA subsequently approved the drug for the treatment of relapsed or refractory B-cell precursor ALL in adults and children.
The EC based its decision on the results of the phase I/II ‘205 clinical trial, an open-label, multicenter, single-arm study of 93 pediatric patients. Participants received intravenous blinatumomab in 28-day cycles: 5 μg/m2 daily for the first seven days and 15 μg/m2 daily for the remaining 21 days, followed by two weeks off treatment, and subsequent cycles of 15 μg/m2 daily for 28 days. Twenty of the 70 patients (28.6%) receiving the recommended dosage achieved a complete response or complete response with partial hematologic recovery within two treatment cycles (p value not provided).
Adverse events (AEs) seen in the study of pediatric patients were similar to those seen in studies of adult patients. The most frequently reported serious AEs were pyrexia, febrile neutropenia, cytokine release syndrome, sepsis, device-related infection, overdose, convulsion, respiratory failure, hypoxia, pneumonia, and multi-organ failure.