New FDA Approval in Acute Myeloid Leukemia Treatment: Onureg

The United States Food and Drug Administration (FDA) on 1 September 2020 approved, for patients diagnosed with acute myeloid leukemia as continuing treatment, Onureg (azacitidine tablets). The patient group specified for this approval consists of patients diagnosed with acute myeloid leukemia who were treated with high-dose chemotherapy and could not complete this treatment, and who achieved their first complete remission after chemotherapy or complete remission without full recovery of blood counts.

The clinical trial leading to this approval (QUAZAR) was a multicenter, randomized, placebo-controlled study. Among the 472 patients enrolled, 238 were treated with Onureg and 234 were assigned to the placebo group. Median overall survival was 24.7 months in patients receiving Onureg and 14.8 months in the placebo group. 

Observed in more than 10% of patients treated with Onureg, the side effects were nausea, vomiting, diarrhea, fatigue, constipation, pneumonia, abdominal pain, joint pain (arthralgia), reduced appetite, febrile neutropenia, dizziness and pain in the extremities.

The FDA gave this study priority review and also granted Onureg orphan drug designation. “Orphan Drug Status” is an FDA program supporting drugs developed to treat relatively rare diseases affecting fewer than 200 thousand people per year in the United States.