FDA Approves Vyxeos for Poor-Prognosis Acute Myeloid Leukemia

Treatment combines daunorubicin and cytarabine

The FDA has approved a combination of daunorubicin and cytarabine (Vyxeos, Jazz Pharmaceuticals) for the treatment of adults with two types of acute myeloid leukemia (AML): newly diagnosed therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC).

The combination of two commonly used chemotherapy drugs into a single formulation may help some patients live longer than if they were to receive the two therapies separately, the agency says.

AML is a rapidly progressing cancer that forms in the bone marrow and results in an increased number of white blood cells in the bloodstream. The National Cancer Institute estimates that approximately 21,380 people will be diagnosed with AML this year; approximately 10,590 patients will die of the disease in 2017.

T-AML occurs as a complication of chemotherapy or radiation in approximately 8% to10% of patients treated for cancer within an average of five years after treatment. AML-MRC is characterized by a history of certain blood disorders and other significant mutations within cancer cells. Patients with t-AML or AML-MRC have very low life expectancies.

The safety and efficacy of Vyxeos were studied in 309 patients with newly diagnosed t-AML or AML-MRC who were randomized to receive Vyxeos or separately administered treatments of daunorubicin and cytarabine. Patients who received Vyxeos lived longer than patients who received separate treatments of daunorubicin and cytarabine (median overall survival, 9.56 months versus 5.95 months).

Common side effects of Vyxeos include hemorrhage, febrile neutropenia, rash, edema, nausea, mucositis, diarrhea, constipation, musculoskeletal pain, fatigue, abdominal pain, dyspnea, headache, cough, decreased appetite, arrhythmia, pneumonia, bacteremia, chills, sleep disorders, and vomiting.

Patients who have a history of serious hypersensitivity to daunorubicin, cytarabine, or any component of the formulation should not use Vyxeos. Patients taking Vyxeos should be monitored for hypersensitivity reactions and decreased cardiac function. Vyxeos has been associated with serious or fatal bleeding events. Daunorubicin has been associated with extravasation. Women who are pregnant or breastfeeding should not take Vyxeos, because it may cause harm to a developing fetus or a newborn baby.

The prescribing information for Vyxeos includes a boxed warning not to interchange Vyxeos with other daunorubicin- and/or cytarabine-containing products.

The FDA granted this application priority review, breakthrough therapy, and orphan drug designations.

Source: FDA; August 3, 2017.