Nilotinib in chronic myeloid leukemia: clinical value, real-world challenges, and the potential of nilotinib tablets to improve therapeutic outcomes.

INTRODUCTION: Nilotinib has been shown to be highly effective for the treatment of chronic myeloid leukemia (CML) and advancing patients to treatment-free remission (TFR). However, its efficacy and safety may be compromised by nonadherence due to its twice-daily fasting requirement. A reduced-dose nilotinib tablet may address these limitations. AREAS COVERED: PubMed search (1966 to July 2026) for papers related to the pathology, etiology, and treatment of CML, pivotal clinical trials and real-world studies for tyrosine kinase inhibitors (TKI) focusing on nilotinib, and recent pharmacokinetic and adherence research, including data on the nilotinib tablet. Nilotinib achieves more rapid deep molecular responses and TFR rates vs imatinib. Nilotinib also offers efficacy comparable to dasatinib, bosutinib, and asciminib. Twice-daily administration with fasting requirements for nilotinib contribute to real-world nonadherence, which often remains undetected by healthcare professionals, undermines TFR goals, and may increase the risk of cardiovascular adverse events. EXPERT OPINION: The nilotinib tablet provides bioequivalent exposure to nilotinib capsules under overnight fasted conditions at a reduced dose without a food effect on absorption. It offers a solution to the complex dosing regimen for nilotinib capsules, potentially supporting better adherence and outcomes with reduced concern about food-related inconvenience.