Tovecimig (CTX-009)

Second-line treatment selection in biliary tract cancer depends on molecular profiling, because approved second-line targeted options are restricted to IDH1 mutations, FGFR2 fusions, MSI-H or dMMR tumours, and BRAF V600E tumours, and the Pan-Asian adapted ESMO guideline recommends molecular analysis in advanced disease considered suitable for systemic treatment. The economic return on that testing is limited by yield: in 91 biliary tract tumours profiled at a single centre, comprehensive genomic profiling produced potentially actionable findings in 23.1% and genomically matched therapy in 7.7%. The cost of failing to establish a primary site is measurable. Among 221 participants with cholangiocarcinoma initiating pemigatinib, those with a prior cancer-of-unknown-primary diagnosis had higher ambulatory resource use (8.2 versus 5.5 visits per patient per month), higher ambulatory costs ($8,584 versus $5,308), mean medical costs of $13,444 versus $9,881 per patient per month, and shorter median overall survival (10.2 versus 30.7 months); the authors conclude that reflexive genomic testing should be encouraged for all participants with cancer of unknown primary.