FDA Approves First Treatment for High-Risk Acute Myeloid Leukemia

Vyxeos is indicated to treat newly diagnosed therapy-related AML or AML with myelodysplasia-related changes.

Today, the FDA approved Vyxeos to treat adults with newly diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC), according to a press release.

Vyxeos is a fixed-combination drug containing daunorubicin and cytarabine. It is the first treatment approved for patients with this form of cancer.

The approval was based on safety and efficacy clinical data of 309 patients with newly diagnosed t-AML or AML-MRC who were randomized to receive either Vyxeos or separately administered daunorubicin and cytarabine. The primary endpoint was overall survival.

The results of the study showed patients in the Vyxeos arm lived longer than those who received separate treatments of daunorubicin and cytarabine. The median overall survival was 9.56 months versus 5.95 months, respectively.

“This is the first approved treatment specifically for patients with certain types of high-risk AML,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, acting director of the FDA’s Office of Hematology and Oncology Products, said in the release. “Vyxeos combines 2 commonly used chemotherapies into a single formulation that may help some patients live longer than if they were to receive the 2 therapies separately.”

The most commonly reported adverse events of Vyxeos were 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 with a history of serious hypersensitivity to daunorubicin, cytarabine, or any component of the formulation should not use Vyxeos, the FDA advises.

An estimated 21,380 individuals will be diagnosed this year with AML, according to the National Cancer Institute. Approximately 10,590 patients with AML will die from the disease in 2017.

T-AML is a complication of chemotherapy or radiation and occurs in approximately 8% to 10% of cancer patients within an average of 5 years of treatment. AML-MRC is characterized by a history of certain blood disorders and other significant mutations within cancer cells, according to the release. Patients with t-AML or AML-MRC have low life expectancies.