Cholangiocarcinoma

On May 28, 2021, the FDA accelerated the approval of infigratinib (Truseltiq; QED Therapeutics), an oral kinase inhibitor, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma (CCA) and FGFR2 fusion or other rearrangement, as detected by an FDA-approved test. The FDA granted infigratinib priority and fast-track review, as well as orphan drug designation for this indication.

Cholangiocarcinoma (CCA) represents a group of heterogeneous cancers that originate in the bile ducts that connect the liver and gallbladder to the small intestine. Although the exact prevalence of CCA is unknown, CCA is a rare cancer; approximately 8000 new cases of CCA are diagnosed annually in the United States.

On January 15, 2021, experts in the management of patients with cholangiocarcinoma (CCA) convened for a virtual accredited continuing education satellite symposium held during the 2021 annual meeting of the American Society of Clinical Oncology Gastrointestinal Cancers Symposium. The goal was to educate healthcare providers on various aspects of CCA, including epidemiology, current standards of care, unmet clinical needs, the safety and efficacy of fibroblast growth factor receptor (FGFR) inhibitors as second-line therapy, and practical approaches to incorporating FGFR inhibitors into the treatment paradigm for the disease.

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 to the ivosidenib group, the difference in median OS was statistically significant.

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.

Targeted therapy has improved survival for patients with cancer across a broad spectrum of disease sites, but until recently, progress has been slow in the treatment of patients with cholangiocarcinoma (CCA).

The first confirmed case in the United States of the 2019 novel coronavirus (COVID-19) was reported on January 21, 2020, and as of November 27, 2020, there were >63 million confirmed cases and >1.5 million deaths globally. Despite the global impact, certain populations have been identified as having a higher risk of developing severe COVID-19 infection, including patients with cancer. Various studies have shown that patients with cancer experience particularly poor outcomes after COVID-19 infection. As a result of the pandemic, care delivery has been disrupted to some degree based on the need for prioritization and limitations on resources. Therefore, healthcare providers and patients have been continually reassessing the balance between the benefits and risks of anticancer interventions in the context of the added risk of COVID-19 infection.

In April 2020, the FDA granted accelerated approval to pemigatinib (Pemazyre), the first targeted therapy for cholangiocarcinoma (CCA). The FGFR inhibitor was approved for adults with CCA and FGFR2 fusion.

Targeted therapy has improved survival for patients with cancer across a broad spectrum of disease sites, but until recently, progress has been slow in applying the use of targeted therapies in the treatment of patients with cholangiocarcinoma (CCA).

Cholangiocarcinoma (CCA) is a rare type of cancer that arises from the intrahepatic or extrahepatic biliary ductal epithelium, accounting for approximately 10% to 15% of all primary hepatic malignancies. CCA is classified as intrahepatic or extrahepatic CCA. In the United States, the incidence of CCA has been rising consistently since 1973.