Onureg Approved for Continuous AML Treatment

Earlier this week, the FDA approved oral azacitidine (Onureg) for patients with acute myeloid leukemia (AML). According to the ASCO Post, Onureg continuously treats adult patients who previously achieved remission. As a result, Onureg offers an opportunity for sustained survival in an effective, accessible, and convenient way. This therapy will be used for patients who cannot partake in intensive curative therapy options, like stem cell transplants, and offers an additional form of continued treatment.


Onureg received FDA approval following data from the Phase 3 QUAZAR AML-001 trial. During this trial, 472 patients received either Onureg or a placebo. To be eligible to participate, patients:

  • Were 55 years old or older
  • Had AML
  • Were within 4 months of remission following chemotherapy
  • Could not be eligible for hematopoietic stem cell transplant (HSCT)

Of the participants, approximately 50.4% received 300mg Onureg daily for a 2-week period, out of a 4-week cycle. The other 49.6% received a placebo. Most patients received 12 cycles of Onureg, versus 6 cycles of the placebo.

Researchers discovered that the treatment improved survival rates by approximately 10 months. Additionally, the therapy seems safe and somewhat well-tolerated. However, there were some adverse reactions, including fetal toxicity, neutropenia, thrombocytopenia, pneumonia, nausea, vomiting, diarrhea, fatigue, constipation, abdominal pain, low appetite, and dizziness. Only one patient experienced a fatal reaction (sepsis). Doctors recommend that patients with myelodysplastic syndromes (MDS) do not take Onureg.

Acute Myeloid Leukemia (AML)

Acute myeloid leukemia (AML) is a blood and bone marrow cancer. In this form, abnormal blood cells develop in bone marrow. These can be myeloblasts (white blood cells), platelets, or red blood cells. As a result of these abnormal cells, healthy cells are crowded out of the bone marrow. The blood then has difficulty doing its job in the body. Radiation and chemical exposure are risk factors for developing AML. Symptoms include:

  • Fever
  • Fatigue
  • Pale skin
  • Easy bruising and bleeding
  • Frequent nosebleeds
  • Periodontal bleeding
  • Difficulty breathing
  • Bone and muscle pain
  • Frequent infections

Learn more about AML.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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