Vyxeos approval a 'welcome therapeutic advance for AML patients'

Vyxeos (daunorubicin and cytarabine) has been approved by the U.S. Food and Drug Administration (FDA). It is the first treatment approved for patients with certain types of high-risk acute myeloid leukemia (AML). The drug, developed by Jazz Pharmaceuticals, is a fixed combination of two chemotherapy drugs, daunorubicin, and cytarabine, and was granted Priority Review, Breakthrough Therapy and Orphan Drug designations by the FDA.

AML is a rapidly progressing cancer that can prove fatal within months. The National Cancer Institute at the National Institutes of Health estimates more than 21,000 people will be diagnosed with the condition this year, with 10,000 people expected to die of the disease in 2017. The condition starts in the bone marrow and usually progresses quickly into the patient's blood and sometimes into other parts of the body, such as the lymph nodes, spleen, liver, and central nervous system. 

Chemotherapy is the main treatment for patients with AML and is sometimes used alongside a targeted therapy drug, such as Rydapt (midostaurin) or Idhifa (enasidenib). Surgery, stem cell transplants, and radiation therapy may also be considered as treatment options. 

Richard Pazdur MD, director of the FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, commented: "Vyxeos combines two commonly used chemotherapies into a single formulation that may help some patients live longer than if they were to receive the two therapies separately."

The drug's safety and efficacy were shown in a study of 309 patients with newly diagnosed t-AML or AML-MRC, each of whom was randomly administered with either Vyxeos or treatments of daunorubicin and cytarabine. The clinical trial showed patients given Vyxeos lived an average of 9.56 months, significantly longer than those with daunorubicin and cytarabine treatments, who survived an average of 5.95 months. 

In a press release from Jazz Pharmaceuticals, Jeffrey E. Lancet MD, chair of the Department of Malignant Hematology at Moffitt Cancer Center, stated: "Vyxeos is the first chemotherapy to demonstrate an overall survival advantage over the standard of care in a Phase 3 randomized study of older adults with newly-diagnosed therapy-related AML or AML with myelodysplasia-related changes… The prognosis for these patients is poor, so the FDA approval of this new drug provides a welcome therapeutic advance."

Side Effects of Vyxeos

Common side effects of Vyxeos include: fever with low white blood cell count, bleeding events, rashes, swelling of tissues, inflammation of the mucous membranes, diarrhea, constipation, musculoskeletal pain, abdominal pain, fatigue, shortness of breath, cough, headaches, decreased appetite, lung infections, abnormal heart rhythm, blood infection, chills, vomiting, and sleep disorders.

Who should not take Vyxeos?

  • Patients with a history of serious hypersensitivity to daunorubicin, cytarabine or any other component of the formulation. 

  • Women who are pregnant or breastfeeding. 

  • Patients taking Vyxeos should be monitored for hypersensitivity reactions and decreased cardiac functions. 

For more information on Vyxeos, visit the link below. 

https://vyxeos.com