The Lynx Group
Cholangiocarcinoma News

Ivosidenib Approved for Advanced or Metastatic Cholangiocarcinoma

Web Exclusives — August 30, 2021

The FDA has approved ivosidenib for adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma (CCA) with an isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.

The FDA also approved the Oncomine Dx Target Test as a companion diagnostic device to assist in selecting patients with CCA for treatment with ivosidenib.

The approval was based on results from a phase 3, multicenter, randomized, double-blind, placebo-controlled trial in which 185 adult patients with locally advanced or metastatic CCA with an IDH1 mutation, who had received 1 to 2 prior treatment regimens (including ≥1 gemcitabine- or 5-fluorouracil–containing regimens), were treated with once-daily 500-mg ivosidenib or placebo until disease progression or unacceptable toxicity.

For the trial, the primary end point was progression-free survival (PFS) as determined by an independent review committee according to RECIST version 1.1. The results indicated a statistically significant improvement in PFS for patients randomized to ivosidenib (hazard ratio [HR], 0.37; 95% confidence interval [CI], 0.25-0.54; P <.0001). The analysis of overall survival was not significant (HR, 0.79; 95% CI, 0.56-1.12; P = .093). Seventy percent of patients randomized to placebo had crossed over to receive ivosidenib after radiographic disease progression.

In a news release from Servier Pharmaceuticals that accompanied the approval of ivosidenib, CCA News editorial advisory board member Rachna T. Shroff, MD, Associate Professor of Medicine, University of Arizona, and Chief of GI Medical Oncology at the University of Arizona Cancer Center, said that, in addition to having an acceptable safety profile, ivosidenib “demonstrated an impressive, significant benefit in progression-free survival, underscoring its importance as a new option for patients battling this aggressive cancer.”

The most common adverse reactions (occurring in ≥15% of patients with CCA) were fatigue, nausea, abdominal pain, diarrhea, cough, decreased appetite, ascites, vomiting, anemia, and rash.

Related Items

FDA Grants TT-00420, a Multi-Kinase Inhibitor, Fast-Track Review for Cholangiocarcinoma
Web Exclusives
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.
Hot Topics in Cholangiocarcinoma and Biliary Tract Cancers: ASCO Highlights
September/October 2021, Vol 2, No 3
At the CCA Summit during the 2021 Annual Meeting of the American Society of Clinical Oncology (ASCO), Mitesh J. Borad, MD, Associate Professor of Medicine, Mayo Clinic, Phoenix, AZ, reviewed some of the key topics presented at ASCO 2021 related to cholangiocarcinoma (CCA) and other biliary tract cancers. Before turning to the specific presentations, Dr Borad welcomed the May 28, 2021, FDA approval of infigratinib (Truseltiq) for patients with advanced or metastatic CCA and FGFR2 fusions or rearrangements. Infigratinib follows pemigatinib (Pemazyre) as the second targeted therapy now available for this patient population.
Important Role of Screening for Genomic Alterations in Cholangiocarcinoma
September/October 2021, Vol 2, No 3
Comprehensive molecular profiling has demonstrated a diverse landscape of oncogenic genomic alterations in cholangiocarcinoma (CCA), which are often the drivers of CCA. In a recent review article, Tanios S. Bekaii-Saab, MD, FACP, Vice Chair and Section Chief for Medical Oncology, Department of Internal Medicine, Mayo Clinic Cancer Center, Phoenix, AZ, and colleagues provided an overview of the molecular heterogeneity of CCA, discussing the role of molecular tests for the diagnosis of patients with intrahepatic CCA, and the implications of the genomic alterations in the treatment of patients with this aggressive disease.
Tibsovo First Targeted Therapy FDA Approved for Advanced or Metastatic CCA with IDH1 Mutation
September/October 2021, Vol 2, No 3
On August 25, 2021, the FDA approved ivosidenib (Tibsovo; Servier Pharmaceuticals), an oral IDH1 inhibitor, for the treatment of adults with previously treated, locally advanced or metastatic cholangiocarcinoma (CCA) and an IDH1 mutation, as detected by an FDA-approved test. The FDA granted ivosidenib a priority review for this indication and an orphan drug designation.
Role of Intraductal Treatment and Endoscopic Oncologists in the Management of Unresectable Extrahepatic Cholangiocarcinoma
September/October 2021, Vol 2, No 3
Although cholangiocarcinoma (CCA) is a rare cancer that originates in the bile ducts, its incidence rate continues to rise in the United States, and many patients are diagnosed late, with unresectable tumor and poor prognosis. The majority of patients with extrahepatic CCA, including the perihilar subtype, require referral to a center with expertise in endoscopic retrograde cholangiopancreatography (ERCP) and interventional radiology, because of the complexities in obtaining a definitive diagnosis and durable biliary drainage.
Role of Radiology in Intrahepatic Cholangiocarcinoma Addressed at 2 Interventional Oncology and Radiology Meetings
September/October 2021, Vol 2, No 3
Key topics related to intrahepatic cholangiocarcinoma (CCA) were presented at the 2021 Society of Interventional Oncology (SIO) and the Society of Interventional Radiology (SIR) meetings and were discussed at the CCA Summit. Bruno C. Odisio, MD, FSIR, Interventional Radiologist and Co-Director of Research, Interventional Radiology, M.D. Anderson Cancer Center, Houston, TX, reviewed the findings.
Recent Developments in Genomic-Driven Therapies for Cholangiocarcinoma
June/July 2021, Vol 2, No 2
Personalized medicine has expanded the treatment options for patients with cholangiocarcinoma (CCA). At the 2021 Annual Meeting of the Cholangiocarcinoma Foundation (CCF), Ghassan K. Abou-Alfa, MD, MBA, Professor of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, discussed recent developments in personalized therapies, highlighting genomic alterations that are informing the new therapies for patients with CCA.
FDA Grants Accelerated Approval to Infigratinib for Metastatic Cholangiocarcinoma
June/July 2021, Vol 2, No 2
The FDA granted accelerated approval to the kinase inhibitor infigratinib (Truseltiq) for the treatment of adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma (CCA) that harbors an FGFR2 fusion or other rearrangement.
Futibatinib in Intrahepatic Cholangiocarcinoma with FGFR2 Fusions or Rearrangements: Primary Results of FOENIX-CCA2 Presented at AACR 2021
June/July 2021, Vol 2, No 2
The primary results of the phase 2 FOENIX-CCA2 clinical trial of futibatinib in patients with previously treated intrahepatic cholangiocarcinoma (CCA) and FGFR2 fusions or rearrangements were presented by Lipika Goyal, MD, MPhil, Assistant Professor of Medicine, Massachusetts General Hospital, Boston, at the 2021 Annual Meeting of the American Association for Cancer Research (AACR).
Truseltiq (Infigratinib) New Targeted Therapy FDA Approved for Advanced or Metastatic Cholangiocarcinoma Harboring FGFR2 Alterations
By Loretta Fala
June/July 2021, Vol 2, No 2
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.

Subscribe to CCA News

Stay up to date with personalized medicine by subscribing to receive the free CCA News print publication or weekly e‑Newsletter.

I'd like to receive: