The European Medicines Agency (EMA) has accepted Otsuka Pharmaceutical and Astex Pharmaceuticals’ marketing authorisation application (MAA) for an oral fixed-dose combination of decitabine and cedazuridine (ASTX727) for the initial treatment of adults with acute myeloid leukaemia (AML) who are not candidates for standard induction chemotherapy.

The application is supported by positive results from the phase 3 ASCERTAIN clinical trial which demonstrated an exposure equivalence of an oral fixed-dose of decitabine and cedazuridine to a standard 5-day regimen of intravenous (IV) decitabine – the current standard of care for AML – using a two-cycle cross-over study design.

Safety findings for the fixed-dose combination of decitabine and cedazuridine were ‘generally consistent’ with those anticipated for IV decitabine, Otsuka reported.

AML is the most common form of acute leukaemia in adults, with a median age at diagnosis of approximately 70 years. In Europe, the incidence of AML is increasing, rising from 3.48 in 1976 to 5.06 patients per 100,000 population in 2013.

The outlook for patients diagnosed with AML has improved over time due to improved care and treatment, but between 2000 and 2007, five-year survival for patients was just 17%, Otsuka reported.

If approved, oral decitabine and cedazuridine would be the first and only oral hypomethylating agents 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.

Oral decitabine and cedazuridine fixed-dose combinations are approved in the US and Canada under the brand name Inqovi for the treatment of adult patients with intermediate and high-risk myelodysplastic syndromes (MDS), including chronic myelomonocytic leukaemia (CMML).