Azacitidine tablets (CC-486) have been approved by the European Commission as a maintenance therapy for AML

On June 18, 2021, it was announced that the European Commission (EC) granted full marketing authorization for azacitidine tablets (CC-486) as a maintenance therapy in adult patients with acute myeloid leukemia (AML). This includes patients who have achieved complete remission or complete remission with incomplete blood count recovery following induction therapy with or without consolidation treatment and who are not eligible for, or choose not to proceed to, hematopoietic stem cell transplantation.¹

This European Commission marketing authorization approval is valid in all 27 member states of the European Union, as well as Iceland, Liechtenstein, and Norway. It follows the Committee for Medicinal Products for Human Use (CHMP) recommendation and the approval by the U.S. Food and Drug Administration (FDA).

The approvals are based on results from the phase III QUAZAR AML-001 international, randomized, double-blind trial investigating CC‑486 maintenance versus placebo in patients aged ≥55 years and with newly diagnosed AML. Study participants had intermediate or poor cytogenetics.

Key results

• Primary endpoint: median overall survival from time of randomization was >2 years (24.7 months) in the CC‑486 arm compared with 14.8 months for the placebo arm (p = 0.0009).
• Median relapse-free survival was significantly longer with CC‑486 than with placebo (10.2 months and 4.8 months, respectively; p < 0.001).
• Adverse events (AEs):
  • Serious adverse reactions occurred in 15% of patients who received CC-486.
  • The most common AEs in both treatment arms were Grade I or II gastrointestinal events, with the following AEs observed in the CC-486 and placebo arms, respectively: nausea (65%; 24%), vomiting (60%; 10%), diarrhea (50%; 21%), fatigue/asthenia (44%; 25%), constipation (39%; 24%), pneumonia (27%; 17%), abdominal pain (22%; 13%), arthralgia (14%; 10%), decreased appetite (13%; 6%), febrile neutropenia (12%; 8%), dizziness (11%; 9%), and pain in extremity (11%; 5%).
  • In patients who received CC-486, common AEs were new or worsening Grade III or IV neutropenia and thrombocytopenia (49% and 22%, respectively).

Read the AML Hub summary here and view the visual abstract of the QUAZAR AML-001 study, which both highlight the efficacy and safety results.