New Drug Onureg® (azacitidine) Approved for Adults with Acute Myeloid Leukemia

The U.S. Food and Drug Administration (FDA) has approved Onureg® (azacitidine tablets) for adults 18 years and older with acute myeloid leukemia (AML). The drug is for use in adults who had a first complete remission (CR) following intensive induction chemotherapy, with or without recovery of blood cell counts, and who are unable to complete intensive curative therapy.¹

Onureg was granted Priority Review, which is granted to new drugs that are very promising and could make a significant impact on the treatment of a serious condition. These programs help drugs like Onureg become available sooner.¹

How does Onureg work?

Onureg is a type of drug called a hypomethylating agent. It works by turning off a protein called DNA methyltransferase. This then turns on genes that stop cancer cells from growing and dividing. This reduces the number of abnormal blood cells and helps control cell growth.²

Why is this an important approval for acute myeloid leukemia?

There is a significant need for medicines that help people with AML survive longer. Research shows:³

• There will be about 20,000 new cases of AML in the United States this year
• Almost 65,000 people in the United States have AML
• AML is a common leukemia
• AML can worsen quickly and can lead to death if not treated quickly
• Many people who receive AML treatment get better and then relapse

This approval provides another treatment option for those with AML for whom other treatments have not been effective.

Evidence for Onureg

The approval of Onureg was based on the results from a phase 3 clinical trial. The trial included 472 people with AML who received either Onureg or a placebo (inactive pill).²

People who took Onureg had a median survival of 24.7 months. People who took a placebo had a median survival of 14.8 months. People who took Onureg lived about 10 months longer than people who took a placebo.²

What are the possible side effects of Onureg?

The most common side effects of Onureg include:⁴

• Nausea and vomiting
• Diarrhea or constipation
• Fatigue or weakness
• Pneumonia
• Stomach pain
• Joint pain
• Decreased appetite
• Pain in the arms or legs

The most frequent severe side effects include:⁴

• New or worsening low white blood cell counts (neutropenia)
• New or worsening low platelet counts (thrombocytopenia)

These are not all the possible side effects of Onureg. Talk to your doctor about what to expect or if you experience any changes that worry you.

Things to know about Onureg

Onureg may be harmful to a developing baby. Women of childbearing age should use effective birth control during treatment with Onureg and for at least 6 months after the final dose. Prior to using this drug, tell your doctor if you are pregnant or plan to become pregnant. Women should not breastfeed during treatment with Onureg and for 1 week after the final dose.⁴

Tell your doctor about any other drugs, vitamins, or supplements you are taking. This includes over-the-counter drugs.

For more information, read the full prescribing information of Onureg.