Data from the phase 3 ASCERTAIN trial support the European Commission’s approval of oral decitabine and cedazuridine as a treatment for those with newly diagnosed acute myeloid leukemia.
The European Commission has approved oral decitabine and cedazuridine (Inqovi) for treating adult patients with newly diagnosed acute myeloid leukemia (AML) who are unable to receive standard induction chemotherapy, according to a press release from Otsuka Pharmaceutical Europe Ltd. and Astex Pharmaceuticals, Inc.1
Supporting data for the European approval came from the phase 3 ASCERTAIN trial (NCT03306264). In the trial, oral decitabine and cedazuridine demonstrated pharmacokinetic exposure equivalence to intravenous decitabine administered across a standard 5-day schedule, thereby meeting the study’s primary end point. Additionally, the safety profile of the fixed-dose oral combination was comparable with that of intravenous decitabine.
In the open-label phase 3 ASCERTAIN trial, patients with myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia, and AML were randomly assigned to one of 2 treatment arms. In the experimental arm, patients received 100 mg of cedazuridine plus 35 mg of decitabine orally as part of 28-day cycles. In the comparator arm, patients received 20 mg/m2 of intravenous decitabine daily.
The European Medicines Agency accepted the marketing authorization application for oral decitabine and cedazuridine in AML not eligible to be treated with induction chemotherapy in August 2022.2
The FDA approved oral decitabine and cedazuridine in adult patients with MDS in July 2020.3 Supporting data for this approval came from the phase 1/2 ASTX727-01-B trial (NCT02103478) and the ASCERTAIN trial.