Combination treatment for AML closer to approval following EMA nod

The European Medicines Agency (EMA) has granted a Marketing Authorisation Application (MAA) for a combination treatment targeting adults with acute myeloid leukaemia (AML) who are not candidates for standard induction chemotherapy. 

The MMA was granted to Otsuka Pharmaceutical and Astex Pharmaceuticals’ oral fixed-dose combination of decitabine and cedazuridine (ASTX727).  

The current standard of care for AML is hospital-administered intravenous (IV) chemotherapy infusions or parenterally administered hypomethylating agents, with treatment cycles typically extending for a week or more. 

ASTX727 is an orally administered, fixed-dose combination of the approved anti-cancer DNA hypomethylating agent, decitabine, together with cedazuridine, an inhibitor of cytidine deaminase (CDA). 

If approved, ASTX727 would be the first and only oral hypomethylating agent licensed in the European Economic Area (EEA) for the initial treatment of adults with AML who are ineligible for intensive chemotherapy, offering a potentially more convenient treatment regimen. 

The MAA is supported by results from a Phase III clinical trial investigating the pharmacokinetic (PK) exposure equivalence of the novel oral fixed-dose combination versus IV decitabine. Orally administered decitabine and cedazuridine showed an exposure equivalence to a standard five-day regimen of IV decitabine.1 

ASTX727 was granted orphan drug designation by the European Commission in December 2021 and in April this year, the EMA agreed to a Paediatric Investigation Plan (PIP) in the EU for the oral decitabine and cedazuridine fixed-dose combination. 


1: Geissler K, Koristek Z, Bernal del Castillo T, et al. Pharmacokinetic exposure equivalence and preliminary efficacy and safety from a randomised crossover Phase 3 study of an oral hypomethylating agent, ASTX727 (DEC-C), compared to IV decitabine in AML patients. Poster presented at EHA Annual Meeting, Vienna, Austria June 9 – 12, 2022. Abstract P573.

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