Despite a major positive impact of proteasome inhibitors (PI), such as bortezomib and carfilzomib, on the survival of patients with multiple myeloma (MM) over the last few years, their use in clinical practice is limited by the development of drug resistance, significant side-effects or constraining administration schedules. Ixazomib is the first, and for now the only, oral PI, which was approved by the US Food and Drug Administration in 2015 and by the European Medicines Agency in 2016. Areas covered: In this review, we provide an overview of the preclinical and early-phase studies of ixazomib used as single-agent and in combination. Furthermore, we discuss the results of a recently published pivotal trial, which evaluated the safety profile and clinical benefit of the combination of ixazomib, lenalidomide and dexamethasone versus lenalidomide and dexamethasone alone in 722 patients with relapsed/refractory MM. Expert opinion: Ixazomib combines the comfort of oral administration, substantial clinical efficacy and a good safety profile with manageable side-effects, which mainly comprise low-grade hematological, digestive or cutaneous events, and the agent will therefore play an active part in long-term treatment strategies, both as single agent and as part of combination regimens. Ongoing phase III trials are currently defining its place in first-line, maintenance and relapse settings.
Keywords: Ixazomib; multiple myeloma; oral administration; proteasome inhibitor.