Otsuka Pharmaceutical Europe has received an amended European Commission marketing authorisation for Inaqovi in combination with venetoclax to treat adults with newly diagnosed acute myeloid leukaemia who are not eligible for standard induction chemotherapy.

The approval expands the use of the oral decitabine and cedazuridine combination following positive Phase 1/2 clinical trial results in patients with limited treatment options.

Acute myeloid leukaemia is the most common form of acute leukaemia in adults and primarily affects people over the age of 60. More than 40,000 people across Europe are estimated to be living with the disease, which continues to have poor long-term survival despite advances in treatment.

The European Commission’s decision is supported by data from the Ascertain-V Phase 1/2 study, which evaluated oral decitabine and cedazuridine alongside venetoclax in adults with newly diagnosed acute myeloid leukaemia who were unsuitable for standard induction chemotherapy.

The study met its primary endpoint, with complete remission achieved in 46.5% of participants after a median of 2.4 months.

Among patients who achieved complete remission, 80% remained in remission after six months and 75.3% after 12 months.

The all-oral combination also demonstrated a median overall survival of 15.5 months. The most common Grade 3 or higher treatment-emergent adverse events were febrile neutropenia, anaemia and neutropenia. Thirty-day and 60-day mortality rates were 3.0% and 9.9% respectively.

Samantha Nier, network director at the Acute Leukemia Advocate Network, said: “Living with acute myeloid leukaemia presents significant challenges for patients and their families, particularly given the poor outcomes often associated with this disease.

“We’re encouraged to see progress that expands treatment options for the AML community, especially for people who have limited choices at diagnosis.”

The approval follows a positive opinion from the Committee for Medicinal Products for Human Use issued in April 2026 and applies across all 27 European Union member states as well as Iceland, Liechtenstein and Norway.

Professor Adriano Venditti, director of the department of onco-haematology at Policlinico Tor Vergata in Rome, said: “Adults with newly diagnosed acute myeloid leukaemia who are not eligible for standard induction chemotherapy face a particularly challenging prognosis and have limited treatment options.

“This approval provides a new option for patients and a valuable addition to the evolving AML treatment landscape.”

Inaqovi combines decitabine with cedazuridine in a fixed-dose oral formulation. Cedazuridine inhibits the breakdown of decitabine in the gut and liver, enabling systemic exposure comparable to intravenous decitabine while allowing treatment to be administered orally.

The amended marketing authorisation provides an additional treatment option for patients unable to receive intensive induction chemotherapy and adds to the growing number of oral therapies available for blood cancers.