2021 Year in Review: Cholangiocarcinoma

Interim results of the multicenter, dose-escalation/dose-expansion study indicate that RLY-4008 is well-tolerated, demonstrating selective targeting of FGFR2 and the potential to overcome FGFR inhibitor resistance in patients with advanced CCA.

Retrospective exposure-response analysis data support 20 mg once daily as the starting dose for futibatinib, whereas exposure-safety analysis for futibatinib demonstrated a significant relationship between hyperphosphatemia and futibatinib exposure.

Results of this retrospective pooled safety analysis indicate that futibatinib was safe and tolerable in patients with advanced solid tumors, including intrahepatic CCA.

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.