The FDA, on August 3, 2017, approved a fixed combination daunorubicin plus cytarabine (Vyxeos) injection for the treatment of adults with 2 types of acute myeloid leukemia (AML)—newly diagnosed, therapy-related AML (t-AML) and AML with myelodysplasia-related changes (AML-MRC). This is the first drug approved by the FDA specifically for t-AML or AML-MRC.
“This is the first approved treatment specifically for patients with certain types of high-risk AML,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence. “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,” he added.
t-AML occurs as a complication of chemotherapy or radiation in 8% to 10% of all patients who receive treatment for any type of cancer, during an average of 5 years after treatment. AML-MRC is a type of AML that results from certain blood disorders that are associated with significant mutations in cancer cells. The prognosis for patients with t-AML or AML-MRC is very short.
This new combination was evaluated in 309 patients with newly diagnosed t-AML or AML-MRC who were randomized to the daunorubicin plus cytarabine combination or to monotherapy with either of the components of the combination. The primary end point was overall survival (OS). The median OS was 9.56 months with the combination versus 5.95 months with monotherapy with either drug.
The most common side effects seen with the combination 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.
This new combination chemotherapy has been associated with serious or fatal bleeding events. Daunorubicin has been associated with localized necrosis. Women who are pregnant or breastfeeding should not use this combination.
The FDA used its priority review process for this indication and granted the new drug a breakthrough therapy designation.