EC Approves Fixed-Dose Oral Combination Treatment for AML

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Otsuka Pharmaceutical Europe and Astex Pharmaceuticals have announced the approval of INAQOVI by the European Commission.

The European Commission (EC) has approved a novel oral fixed-dose combination treatment—INAQOVI (oral decitabine and cedazuridine)—for use in adult patients with newly diagnosed acute myeloid leukemia (AML) who cannot have standard induction chemotherapy.

According to a Sep. 19, 2023 press release, Otsuka Pharmaceutical Europe and Astex Pharmaceuticals’ INAQOVI’s commission approval was based on the results from a Phase III clinical trial (ASCERTAIN), which investigated the pharmacokinetic exposure equivalence of the therapy against intravenous (IV) decitabine in AML patients. The Phase III trial, which included 89 AML patients, successfully met its primary endpoint, demonstrating that the orally administered therapy achieved equivalent pharmacokinetic exposure to that achieved with IV decitabine and both with similar pharmacodynamic activity. The safety of the oral treatment option was considered to be consistent with that of the IV decitabine.

INAQOVI is an oral treatment combining decitabine (35 mg)—an approved hypomethylating agent (HMA)—with cedazuridine (100 mg)—an inhibitor of cytidine deaminase. The therapy works through inhibiting cytidine deaminase in the gut and liver, which allows for systemic exposure to decitabine through oral administration that is comparable to that achieved intravenously over a five-day period. With the EC’s approval, INAQOVI is the first oral therapy that incorporates a hypomethylating agent (decitabine) licensed within the European Economic Area for the treatment of AML patients.


Currently, AML patients are either administered IV chemotherapy infusions in a hospital setting, or administered hypomethylating agents parenterally when chemotherapy is not applicable. An oral option for the treatment of AML can help to improve quality of life for this patient population.

Furthermore, the European Medicines Agency agreed to a Paediatric Investigation Plan for the oral fixed-dose combination in June 2022, which has the potential to lead to clinical trials for the use of this therapy in children with AML.

Source: Astex Pharmaceuticals