Cholangiocarcinoma

Findings of patient-reported outcomes from the phase 2 FOENIX-CCA2 study indicate that quality-of-life data and overall health status were maintained among futibatinib-treated patients with advanced intrahepatic CCA.

Initial results of the phase 2 FOENIX-CCA2 study demonstrated that second-line futibatinib was active and had a manageable safety profile in patients with intrahepatic CCA harboring FGFR2 fusion/rearrangements.

Longitudinal evaluation of quality of life in pemigatinib-treated patients with advanced CCA harboring FGFR fusions/rearrangements showed that those who achieved complete response/partial response or stable disease exhibited stable overall health status and emotional functioning.

Findings from a study in a Chinese patient population confirmed the efficacy and tolerability of pemigatinib in patients with recurrent or metastatic CCA with an FGFR2 fusion or rearrangement.

Updated results from the FIGHT-202 study supported the primary data that second-line pemigatinib treatment resulted in efficacy and sustained tolerability in patients with CCA harboring FGFR2 rearrangements/fusions.

Final results of the ClarIDHy trial demonstrated the clinical benefit of ivosidenib in patients with previously treated unresectable or metastatic CCA harboring an IDH1 mutation.

Mature results from a phase 2 study demonstrated that the FGFR inhibitor infigratinib has antitumor activity and a manageable adverse event profile in previously treated patients with locally advanced or metastatic CCA harboring FGFR2 gene fusions or rearrangements.

Real-world evidence demonstrates a survival advantage for patients with CCA harboring FGFR2 fusions/rearrangements compared with those with FGFR2 wild-type CCA.

On November 3, 2021, TransThera Sciences announced that the FDA granted its investigational drug, TT-00420, a spectrum-selective multi-kinase inhibitor, a fast-track designation for the treatment of patients with cholangiocarcinoma (CCA) who have no available standard treatment options. In preclinical studies, TT-00420 has shown high activity in a variety of FGFR mutations.