FDA Approves Blinatumomab for B-Cell ALL

Roxanne Nelson

December 03, 2014

The US Food and Drug Administration (FDA) today approved blinatumomab (Blincyto, Amgen), a drug with a unique mechanism of action, for use in the treatment of patients with Philadelphia chromosome-negative precursor B-cell acute lymphoblastic leukemia (ALL).

The approved indication is for patients who have relapsed or who were refractory to previous treatment.

Blinatumomab passed through the agency's accelerated approval program, and has been granted breakthrough therapy designation, priority review, and orphan product designation. This designation is used for potentially important new agents, and typically cuts the approval decision time.

In this case, approval has been granted more than 5 months ahead of the prescription drug user fee goal date of May 19, 2015, which is the date that the FDA was scheduled to complete their review of this application.

Blinatumomab is the first of a novel class of drugs known as bispecific T-cell engagers (BiTE) and is designed to direct cytotoxic T-cells to CD19-expressing cancer cells. CD19 is a protein expressed on the surface of B-cell-derived ALLs and non-Hodgkin's lymphomas.

"Immunotherapies, especially [blinatumomab] with its unique mechanism of action, are particularly promising for patients with leukemia," said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA Center for Drug Evaluation and Research, in a statement. "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 FDA based its approval on the results of a clinical trial that evaluated the safety and effectiveness of blinatumomab in a cohort of 185 adults with Philadelphia chromosome-negative relapsed or refractory precursor B-cell ALL. All participants received infusion treatment for at least 4 weeks, and 32% of participants experienced a complete remission for approximately 6.7 months.

As part of the accelerated approval program, the manufacturer (Amgen) is required to conduct a postmarketing study to verify that survival has improved in this patient population.

The most common adverse effects seen in blinatumomab-treated patients were fever, headache, peripheral edema, febrile neutropenia, nausea, hypokalemia, fatigue, constipation, diarrhea, and tremor.

The drugs will have a boxed warning to alert patients and healthcare professionals to the finding that some clinical trial participants experienced problems with low blood pressure and difficulty breathing as a result of cytokine-release syndrome when they began treatment. There were also cases of encephalopathy and other adverse events affecting the central nervous system.

The FDA has approved the agent with a Risk Evaluation and Mitigation Strategy, which consists of a communication plan to inform healthcare providers about the serious risks and the potential for preparation and administration errors.


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