Carfilzomib is a second-generation proteasome inhibitor that binds selectively and irreversibly with the chymotrypsin-like site of the proteolytic core. Its initial approval by the Food and Drug Administration, as monotherapy for relapsed/refractory multiple myeloma (RR-MM), followed soon by a global authorization of its combination with dexamethasone or with lenalidomide plus dexamethasone for the treatment of RR-MM after 1-3 prior lines. In order to optimize its administration, carfilzomib is currently examined in different doses and regimens in relapsed/refractory as well as in newly diagnosed myeloma. Areas covered: This review will focus on the introduction of carfilzomib as an effective anti-myeloma treatment, describing the evolution of the drug from its pre-clinical development to its established use by phase III clinical trials. Based on the latest evidence, we will present its mechanism of action, its efficacy and its toxicity profile on treated myeloma patients and we will try to reply to all raised concerns about its current use. Expert opinion: Either alone or in combination with other agents, carfilzomib seems to be an effective and safe therapeutic option for MM management. Results of ongoing trials are expected to update its application, even at an earlier level of the disease course.
Keywords: Carfilzomib; multiple myeloma; newly diagnosed; proteasome inhibitor; relapsed/refractory.