FDA Approves Blincyto to Treat Acute Lymphoblastic Leukemia

Today the US Food and Drug Administration has approved Blincyto (blinatumomab) for the treatment of patients with Philadelphia chromosome-negative precursor B-cell acute lymphoblastic leukemia (B-cell ALL), a very uncommon form of ALL.

Precursor B-cell ALL is an aggressive type of cancer in which the bone marrow makes too many B-cell lymphoblasts, an immature type of white blood cell. The Philadelphia chromosome is an abnormality that sometimes occurs in the bone marrow cells of leukemia patients. The National Cancer Institute estimates that 6,020 Americans will be diagnosed with ALL and 1,440 will die from the disease in 2014.

Blincyto is an immunotherapy. This means that it is a treatment that uses certain parts of a person’s immune system to fight disease. Blincyto is the first approved drug that engages the body’s T-cells, a type of white blood cell or lymphocyte, to destroy leukemia cells. The drug acts as a connector between a protein called CD19, which is found on the surface of most B-cell lymphoblasts, and CD3, a protein on T-cell lymphocytes. It is intended for patients whose cancer returned after treatment (relapsed) or did not respond to previous treatment (refractory).

"Immunotherapies, especially Blincyto with its unique mechanism of action, are particularly promising for patients with leukemia," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. "Recognizing the potential of this novel therapy, the FDA worked proactively with the sponsor under our breakthrough therapy designation program to facilitate the approval of this novel agent."

The safety and effectiveness of Blincyto were evaluated in a clinical study involving 185 adults with Philadelphia chromosome-negative relapsed or refractory precursor B-cell ALL. All participants were treated with Blincyto for at least four weeks via infusion, a method used to inject treatment into the bloodstream using a needle. Results showed 32 percent of participants had no evidence of disease (complete remission) for approximately 6.7 months.

Blincyto carries a boxed warning alerting patients and health care 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, experienced a short period of difficulty with thinking (encephalopathy) or other side effects in the nervous system. The most common side effects seen in Blincyto-treated participants were fever (pyrexia), headache, swelling of tissues (peripheral edema), fever with a low number of white blood cells (febrile neutropenia), nausea, low potassium (hypokalaemia), fatigue, constipation, diarrhea and tremor.

Source: FDA