Medication Monitor

Generic Name (Trade Name—Company)
March 29, 2018


Approval expanded to treat patients with B-cell precursor ALL at risk for relapse

FDA granted accelerated approval to blinatumomab to treat adults and children with B-cell precursor acute lymphoblastic leukemia (ALL) who are in remission but still have minimal residual disease (MRD) and therefore an increased risk of relapse. 

Blinatumomab works by attaching to CD19 protein on the leukemia cells and CD3 protein found on certain immune system cells. Bringing the immune cell close to the leukemia cell allows the immune cells to attack the leukemia cells better.

FDA first approved blinatumomab under accelerated approval in December 2014 for treatment of Philadelphia chromosome–negative relapsed or refractory positive B-cell precursor ALL. Full approval for this indication was granted in July 2017, and at that time, the indication was also expanded to include patients with Philadelphia chromosome–positive ALL.

Approval was based on results of single-arm clinical trial that included 86 patients in first or second complete remission who had detectable MRD in at least 1 out of 1,000 cells in their bone marrow. Efficacy was based on achievement of undetectable MRD in an assay that could detect at least 1 cancer cell in 10,000 cells after one cycle of treatment with blinatumomab, in addition to the length of time patients remained alive and in remission (hematological relapse-free survival).

Overall, undetectable MRD was achieved by 70 patients. More than one-half of the patients remained alive and in remission for at least 22.3 months.

Adverse effects for this indication are consistent with those seen in other uses of the drug: infections (bacterial and pathogen unspecified), fever, headache, infusion-related reactions, neutropenia, anemia, febrile neutropenia, and thrombocytopenia.

The agent carries a boxed warning alerting patients and health professionals that some clinical trial participants had problems with low blood pressure and difficulty breathing (cytokine release syndrome) at the start of the first treatment, and experienced a short period of difficulty with thinking (encephalopathy) or other adverse nervous effects.

Serious risks include infections, effects on the ability to drive and use machines, pancreatitis, and preparation and administration errors—instructions for preparation and administration should closely be followed. There is a risk of serious adverse reactions in pediatric patients due to benzyl alcohol preservative; therefore, the drug prepared with preservative-free saline should be used for patients weighing less than 22 kg.