EMA Starts Review of Oral Fixed-Dose Combination Treatment for AML

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EMA has accepted the marketing authorization application for an oral fixed-dose combination treatment as an initial treatment for adults with AML.

The European Medicines Agency (EMA) has accepted the marketing authorization application (MAA) for the oral fixed-dose combination treatment (decitabine and cedazuridine [ASTX727]) as an initial treatment for adults with acute myeloid leukemia (AML). The decision was announced in an Aug. 22, 2022 press release from Otsuka Pharmaceutical Europe and Astex Pharmaceuticals.

Currently, the standard of care for patients with AML is hospital-administered intravenous (IV) chemotherapy infusions or parenterally administered hypomethylating agents. If the oral combination treatment of decitabine and cedazuridine is approved, it would become the first and only oral hypomethylating agent licensed in the European Economic Area for the initial treatment of patients with AML who are ineligible for intensive chemotherapy.

Results from the Phase III ASCERTAIN clinical trial, which investigated the pharmacokinetic (PK) exposure equivalence of the combination therapy versus IV decitabine, have provided support for the MAA. The primary endpoint of the study was met, whereby the combination therapy showed exposure equivalence to a standard five-day regimen of IV decitabine, and the safety profile of the combination therapy was generally consistent with the comparator therapy.


In December 2021, the combination therapy was granted orphan drug designation by the European Commission and in April 2022, EMA agreed to a Paediatric Investigation Plan for the combination therapy, which is an important milestone for the further clinical evaluation in children with AML.

Source: Otsuka Pharmaceuticals