Oral decitabine and cedazuridine granted approval by the European Commission for the treatment of adults with newly diagnosed AML

On September 19, 2023, oral decitabine in combination with cedazuridine, a cytidine deaminase inhibitor, was approved by the European Commission for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for standard induction chemotherapy.¹ This approval is based on the favorable data demonstrated in the phase III ASCERTAIN study (NCT03306264) clinical trial, which investigated the pharmacokinetic exposure equivalence of an oral fixed-dose combination of decitabine and cedazuridine versus intravenous (IV) decitabine.¹

Key results of the ASCERTAIN trial¹

The primary endpoint was met, with the oral decitabine and cedazuridine fixed-dose combination demonstrating a pharmacokinetic exposure equivalence of 99.64% (90% confidence interval [CI], 91.23–108.8) to standard 5-day IV administration of 20 mg/m² of decitabine.  

• Median overall survival was 7.9 months (95% CI, 5.9–13.0).
• Complete response rate was 21.8% at 7.95 months’ median follow up.
• Safety profile was consistent with IV decitabine.
  • The most common adverse event (occurring in ≥20% of patients) was thrombocytopenia.
  • The most common serious adverse events (≥20%) were febrile neutropenia and pneumonia.
  • 14% of patients discontinued treatment, including 5% due to pneumonia.