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FDA expands approval of Blincyto for leukemia at risk for relapse
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The FDA expanded the approval of blinatumomab for the treatment of adults and children with B-cell precursor acute lymphoblastic leukemia who are in remission but still have minimal residual disease.
Blinatumomab (Blincyto, Amgen) — a CD19–directed CD3-bispecific T-cell engager immunotherapy — is indicated for patients with both Philadelphia chromosome-positive and Philadelphia chromosome-negative ALL.
The FDA based the expanded approval on a single-arm trial of 86 patients with first or second complete remission of ALL who had detectable minimal residual disease (MRD) in at least one of 1,000 bone marrow cells.
Researchers defined efficacy as achievement of undetectable MRD through an assay that could detect one cancer cell among 10,000 cells after one cycle of treatment with blinatumomab.
Seventy patients achieved undetectable MRD. Fifty percent of patients remained alive and in remission for at least 22.3 months.
“This is the first FDA-approved treatment for patients with MRD-positive ALL,” Richard Pazdur, MD, director of the FDA Oncology Center of Excellence and acting director of the office of hematology and oncology products in the FDA Center for Drug Evaluation and Research, said in a press release. “Because patients who have MRD are more likely to relapse, having a treatment option that eliminates even very low amounts of residual leukemia cells may help keep the cancer in remission longer.”
The safety profile was consistent with the other uses of blinatumomab.
Common side effects included bacterial and pathogen-unspecified infections, pyrexia, headache, infusion-related reactions, neutropenia, anemia, febrile neutropenia and thrombocytopenia.
Blinatumomab carries a boxed warning that some clinical trial participants had problems with cytokine release syndrome — evident through low blood pressure and difficulty breathing — at the start of the first treatment, in addition to other side effects of the nervous system.
“We look forward to furthering our understanding about the reduction in MRD after treatment with Blincyto,” Pazdur said. “Studies are being conducted to assess how Blincyto affects long-term survival outcomes [among] patients with MRD.”
The FDA approved the new indication under the accelerated pathway. Blinatumomab previously received orphan drug designation.