Cholangiocarcinoma

On May 28, 2021, the FDA granted accelerated approval to infigratinib (Truseltiq; QED Therapeutics, Inc), an oral FGFR1-3 selective inhibitor, for the treatment of adults with previously treated locally advanced or metastatic cholangiocarcinoma (CCA) with an FGFR2 fusion or other rearrangement. The FDA previously granted infigratinib orphan drug and fast-track designations. Read More ›

Final results from the phase 3 clinical trial ClarIDHy showed that ivosidenib (Tibsovo), a first-in-class oral inhibitor of isocitrate dehydrogenase 1 (IDH1) mutation, prolonged the median overall survival (OS) in patients with previously treated advanced cholangiocarcinoma (CCA) and IDH1 mutation. Although this improvement did not reach statistical significance, after adjusting for crossovers from the placebo group to the ivosidenib group, the difference in median OS improvement was statistically significant. Read More ›

The recent FDA approval of the first FGFR inhibitor, pemigatinib (Pemazyre), and the positive results from the phase 3 study of the first IDH1 inhibitor, ivosidenib (Tibsovo), represent major breakthroughs in the treatment of patients with cholangiocarcinoma (CCA), a rare cancer associated with poor outcomes. However, the duration of response with these agents is still relatively short. Read More ›

Cholangiocarcinoma (CCA), a type of biliary tract cancer, is a rare malignancy, with no FDA-approved medications specifically for this type of cancer. The current standard first-line treatment for patients with locally advanced or metastatic CCA is a chemotherapy combination of gemcitabine and cisplatin. Read More ›

Alterations in the fibroblast growth factor receptor (FGFR)2 gene have been identified as driver mutations in cholangiocarcinoma (CCA). Durable objective responses were observed in >33% of patients with locally advanced or metastatic CCA and FGFR2 rearrangements or fusions who received treatment with pemigatinib, a selective oral inhibitor of FGFR1, FGFR2, and FGFR3. Data from the single-arm, open-label phase 2 clinical trial FIGHT-202, which was presented at the ESMO Congress 2019, revealed that investigational pemigatinib induced a response in 35.5% of the 107 patients with FGFR2 fusions or rearrangements (cohort A), with a median duration of response of 7.5 months. Read More ›

Ivosidenib (Tibsovo), an oral therapy that targets isocitrate dehydrogenase-1 (IDH1) mutation, significantly improved progression-free survival (PFS) in patients with advanced cholangiocarcinoma (CCA) and an IDH1 mutation, in a phase 3 clinical trial reported lead investigator Ghassan K. Abou­Alfa, MD, Medical Oncologist, Gastrointestinal Oncology Service, Memorial Sloan Kettering Cancer Center, New York City, at the ESMO Congress 2019. Read More ›

Diagnosis of cholangiocarcinoma (CCA) is often identified at a late stage. Analyzing the tumor-specific mutation profile of a patient with CCA can improve the diagnosis and treatment for the individual patient. The molecular profile of CCA can be done through the use of circulating tumor (ct) DNA sequencing, which may help to target specific mutations and improve treatment selection for this rare type of cancer. Read More ›