FDA Approves First Treatment for Certain Types of Acute Myeloid Leukemia

Vyxeos, a fixed-combination of two chemotherapy drugs Daunorubicin and Cytarabine was approved by the U.S. Food and Drug Administration for the treatment of adults with two types of acute myeloid leukemia (AML). “This is the first approved treatment specifically for patients with certain types of high-risk AML,” said Richard Pazdur, M.D., 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. “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 approval of Vyxeos has been granted to Jazz Pharmaceuticals.

Acute Myeloid Leukemia

In acute myeloid leukemia (AML) the bone marrow makes excess amounts of a type of white blood cell called myeloblasts. Although AML starts in the bone marrow it quickly progresses to the bloodstream and is hence termed “acute”. Other blood-forming cells can also become AML. Symptoms of AML include fever, fatigue and easy bleeding.

Risk factors of acquiring AML have been listed as being male, smoking, previous chemotherapy or radiation therapy, prior treatment for childhood acute lymphoblastic leukemia (ALL), exposure to radiation from an atomic bomb and having certain blood disorders. AML can sometimes spread to other parts of the body.

There are different subtypes of AML. This study is concerned with two types of high-risk AML, namely, newly diagnosed therapy-related AML (t-AML) and AML with myelodysplasia-related changes (AML-MRC).

T-AML is cancer that results due to prior exposure to chemotherapy or radiation (mostly given for breast and colon cancers) that occurs within an average of five years after start of treatment, in approximately 8 to10 percent of all treated patients. When patients have AML-MRC they are faced with a history of certain blood disorders and other significant mutations within cancer cells. Patients with either of the conditions have very low life expectancies.

There are four types of standard treatment methods used for AML: Chemotherapy (including Daunorubicin and Cytarabine), Radiation therapy, Stem cell transplant and anticancer drug therapy that includes arsenic trioxide and all-trans retinoic acid (ATRA).

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The National Cancer Institute at the National Institutes of Health has estimated that in 2017 there will approximately be 21,380 people diagnosed with AML and 10,590 deaths due to the disease.

The outcome of patients with AML depends on patient and disease characteristics and the therapy method. 

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Daunorubicin, (or daunomycin), and Cytarabine, (or cytosine arabinoside (ara-C)) are chemotherapy medications specifically used for acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), and chronic myelogenous leukemia (CML). They are used in injection forms in high doses to treat the conditions.

Study

The safety and efficacy of Vyxeos were tested in a randomized study in which patients received either Vyxeos or separately administered treatments of Daunorubicin and Cytarabine. There were 309 patients in total in the study who had been diagnosed with t-AML or AML-MRC for the first time. The trial measured the overall survival of the patients (how long patients lived from the date they started the trial).

The end results showed that patients who received Vyxeos lived longer (median overall survival 9.56 months) than patients who received separate treatments of Daunorubicin and Cytarabine (median overall survival 5.95 months).

Contraindications while using Vyxeos

Although beneficial Vyxeos has its share of side effects that include serious or fatal bleeding events, fever with low white blood cell count, rashes, edema, nausea, diarrhea, constipation, muscle and joint pain, fatigue, chills, abdominal pain, difficulty in breathing, headache, cough, decreased appetite, arrhythmia, pneumonia, and blood infections.

Patients who are prone to be sensitive to Daunorubicin or Cytarabine have been advised not to use Vyxeos.

Also, patients taking Vyxeos should be closely monitored for hypersensitivity reactions and decreased cardiac function.

Patients taking Vyxeos are also warned against interchanging the drug with other Daunorubicin- and/or Cytarabine-containing products.

Vyxeos should not be taken by pregnant or breastfeeding women because it could harm a developing fetus or a newborn baby.

Combinations in general usually work better than single drugs in chemotherapy as they comprise of different drugs that work in different ways. Also, since t-AML and AML-ARC are very high-risk types of acute myeloid leukemias that could be fatal, the move that FDA has made in approving the combination drug Vyxeos to be administered for patients suffering from these conditions will benefit them by prolonging their lives.

References:

1. FDA approves first treatment for certain types of poor-prognosis acute myeloid leukemia - (https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm569883.htm)
2. What Is Acute Myeloid Leukemia? - (https://www.cancer.org/cancer/acute-myeloid-leukemia/about/what-is-aml.html)
3. Adult Acute Myeloid Leukemia Treatment (PDQ®)–Patient Version - (https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq)

Source-Medindia