FDA Grants Priority Review to Blinatumomab for Pediatric ALL

The U.S. Food and Drug Administration (FDA) granted priority review to blinatumomab for the treatment of pediatric and adolescent patients with Philadelphia chromosome-negative (Ph−) relapsed/refractory B-cell precursor acute lymphocytic leukemia (ALL).

The decision was based on the results from a single-arm, multi-center, dose-finding phase I/II efficacy trial that included pediatric patients (<18 years) with Ph− B-cell precursor ALL who were refractory, had relapsed at least twice, or relapsed after an allogeneic hematopoietic cell transplantation (alloHCT).

In the phase I dose-finding portion, an independent Data Safety Monitoring Board recommended stepwise dosing of blinatumomab 5/15-μg/m²/day). In the primary phase II portion evaluating safety and efficacy, patients are being monitored for a primary endpoint of complete remission within the first two cycles of blinatumomab treatment. Secondary endpoints include incidence of adverse events (AEs), the proportion of patients undergoing alloHCT after blinatumomab treatment, relapse-free survival, and overall survival. Minimal residual disease (MRD) response and complete MRD response were exploratory endpoints in both phases.

Among the 70 patients who received the recommended dose of blinatumomab, the most common grade ≥3 AEs included anemia, thrombocytopenia, febrile neutropenia, hypokalemia, and neutropenia. All patients in the study have completed therapy and are being followed for long-term data.

Source: Amgen press release, May 4, 2016.