Inaqovi in combination with venetoclax has received an amended European Commission marketing authorisation for newly diagnosed acute myeloid leukaemia patients ineligible for standard induction chemotherapy.

Otsuka Pharmaceutical Europe has received an amended European Commission marketing authorisation for Inaqovi (oral decitabine and cedazuridine) in combination with venetoclax for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) who are not eligible for standard induction chemotherapy.

The decision applies across all 27 EU member states, as well as Iceland, Liechtenstein and Norway, following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in April 2026.

Acute myeloid leukaemia is the most common form of acute leukaemia in adults and is typically diagnosed in older patients. Despite advances in treatment, outcomes remain poor, with five-year survival in adults estimated at around 17%.

Across Europe, more than 40,000 people are affected by AML, with incidence rising in line with an ageing population.

The approval is supported by data from the phase 1/2 ASCERTAIN-V clinical trial, which evaluated oral decitabine and cedazuridine in combination with venetoclax in patients with newly diagnosed AML ineligible for intensive chemotherapy.

The study met its primary endpoint, with complete remission achieved in 46.5% of participants at a median of 2.4 months. Among patients who achieved complete remission, 80% remained in remission at six months and 75.3% at 12 months.

Median overall survival was reported at 15.5 months. The most frequent Grade 3 or higher adverse events included febrile neutropenia (49.5%), anaemia (38.6%) and neutropenia (35.6%). Thirty- and 60-day mortality rates were 3.0% and 9.9% respectively.

Samantha Nier, network director at the Acute Leukemia Advocate Network, said the decision represents an important step for patients with limited treatment options at diagnosis.

“Living with acute myeloid leukaemia presents significant challenges for patients and their families, particularly given the poor outcomes often associated with this disease,” Nier said. “We’re encouraged to see progress that expands treatment options for the AML community, especially for people who have limited choices at diagnosis.”

Professor Adriano Venditti, director of the Department of Onco-Hematology at Policlinico Tor Vergata in Rome, said the approval adds to the evolving treatment landscape in AML.

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

The oral fixed-dose combination contains decitabine, a hypomethylating agent, and cedazuridine, an inhibitor of cytidine deaminase designed to enable oral administration by increasing systemic exposure.

The approval follows earlier regulatory progress, including a positive CHMP opinion in April 2026.