Otsuka Pharmaceutical's Inaqovi Approved in Europe, Providing an Oral Combination Therapy for Elderly Patients with Acute Myeloid Leukemia
EMA Approval of Inaqovi and a Shift in Treatment Paradigm
The European Medicines Agency (EMA) has approved Inaqovi, developed by Astex Pharmaceuticals, a subsidiary of Otsuka Pharmaceutical, for the treatment of acute myeloid leukemia (AML). Inaqovi is an oral combination therapy consisting of cedazuridine, a cytidine deaminase (CDA) inhibitor, and decitabine, a DNA methyltransferase (DNMT) inhibitor. Previously, elderly and frail patients had to receive treatment via regular intravenous (IV) or subcutaneous (SC) injections at a hospital. Inaqovi offers an innovative alternative that can be taken at home, which will significantly improve patient convenience and healthcare resource efficiency. This represents a significant regulatory milestone.
Demonstrated Pharmacokinetic Equivalence and the Value of Phase 3 Clinical Trials
This approval is based on data from the global Phase 3 ASCERTAIN study. In this study, researchers successfully demonstrated that the pharmacokinetic (PK) exposure levels of the oral Inaqovi group were nearly equivalent to those of the existing intravenous decitabine group. The safety profile was also confirmed to be similar to that of existing intravenous therapy, supporting the clinical efficacy of the oral combination therapy. As a result, it is now possible to expect the same level of epigenetic therapeutic effect while reducing the risk of infection and the burden of hospital visits associated with intravenous therapy.
Expansion of Combination Therapy with Venetoclax, a Standard Treatment
Notably, in June 2026, the European Commission (EC) will additionally approve the combination therapy of Inaqovi and venetoclax. According to current guidelines, the standard of care (SOC) for acute myeloid leukemia patients who are ineligible for chemotherapy is a combination of intravenous hypomethylating agents (HMAs) and venetoclax. With this approval, a complete all-oral treatment regimen, in which the entire treatment schedule can be replaced with oral medications, will become a reality. This will improve the quality of life for patients and strengthen the market competitiveness of the combination therapy, leading to an expansion of Otsuka Pharmaceutical's market share.
Changes in Competitive Landscape and Outlook for the Acute Myeloid Leukemia Market
The acute myeloid leukemia treatment market is expected to grow rapidly at an annual rate of approximately 7% to 10%, reaching approximately $3.1 billion to $4.3 billion by 2026. Inaqovi has the potential to lead a market reshuffle by replacing existing intravenous HMAs such as decagen and vidaza. In particular, by focusing on patients with limited access to hospital treatment, Otsuka Pharmaceutical can establish a dominant position in the area of elderly patients with high unmet medical needs. Although the cost of treatment per cycle is relatively high, ranging from $8,700 to $10,800, this will be overcome through patient support programs and reimbursement.
The European approval of Inaqovi and the expansion of the venetoclax (Venclyxto) combination therapy are expected to mark a turning point in the treatment paradigm for acute myeloid leukemia, shifting from intravenous-centric treatment to a patient-friendly oral combination therapy. Pharmacokinetic equivalence and safety, demonstrated through the global Phase 3 ASCERTAIN study and the Phase 1/2 ASCERTAIN-V trial, are key indicators that will enable Inaqovi to gain an advantage in direct market competition with existing intravenous standard treatments such as vidaza. In the global acute myeloid leukemia market, estimated at approximately $3.1 billion to $4.3 billion by 2026, Inaqovi is expected to contribute to Otsuka Pharmaceutical's long-term diversification of its oncology revenue by leveraging the advantages of its independent oral pipeline. In the short term, the speed of price negotiations and reimbursement approvals in each European country will be crucial for initial market penetration, and in the long term, the all-oral combination therapy with venetoclax is likely to become the standard of care for elderly patients.
Source: EMA (ema)