Turalio risk evaluation and mitigation strategy for treatment of tenosynovial giant cell tumor: framework and experience

Abstract

For drugs with enhanced serious safety risks, Risk Evaluation and Mitigation Strategy (REMS) may be required. Pexidartinib is approved for treatment of adult symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery. Its approval was conditional on its prescription via a mandatory REMS due to serious and potentially fatal liver injury seen in clinical trials. Turalio^® REMS aims to mitigate this risk by ensuring provider education on pexidartinib use and required REMS components, prescriber adherence to baseline and periodic monitoring, and enrolling patients in a registry to further assess safe use and acute, chronic and irreversible hepatotoxicity. Through Turalio REMS, benefits of treating patients with pexidartinib may be preserved.

Keywords: Pexidartinib; REMS framework; Turalio REMS Program; elements to assure safe use; hepatotoxicity; knowledge, attitude and behavior assessment; liver adverse event reporting criteria; risk evaluation and mitigation strategy; risk minimization measures; tenosynovial giant cell tumor.