Ivosidenib has been approved by the FDA for advanced or metastatic cholangiocarcinoma

Sept 19, 2021: The Food and Drug Administration approved Ivosidenib (Tibsovo, Servier Pharmaceuticals LLC) for adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with an isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.

Today, the FDA also approved the Oncomine Dx Target Test (Life Technologies Corporation) as a companion diagnostic device to aid in selecting patients with cholangiocarcinoma for treatment with ivosidenib.

Ivosidenib was investigated in a randomised (2:1), multicenter, double-blind, placebo-controlled clinical trial (Study AG120-C-005, NCT02989857) of 185 adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 mutation. The patient’s disease must have progressed following at least one, but not more than two prior regimens, including at least one gemcitabine- or 5-flurouracil-containing regimen. Patients were randomised to receive either ivosidenib 500 mg orally once daily or matched placebo until disease progression or unacceptable toxicity.

The primary efficacy endpoint was progression-free survival (PFS) as determined by independent review committee according to RECIST 1.1. The trial demonstrated a statistically significant improvement in PFS for patients randomised to ivosidenib (HR 0.37; 95 percent CI: 0.25, 0.54; p<0.0001). The analysis of OS was not significant (0.79; 95 percent CI: 0.56, 1.12; p=0.093); 70 percent of patients randomised to placebo had crossed over to receive ivosidenib after radiographic disease progression.

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

The recommended ivosidenib dosage for cholangiocarcinoma is 500 mg orally once daily with or without food until disease progression or unacceptable toxicity.

Take second opinion on cholangiocarcinoma

Send Details

Susan Hau

Website |  + postsBio ⮌

Susan Hau is a distinguished researcher in the field of cancer cell therapy, with a particular focus on T cell-based approaches and cancer vaccines. Her work spans several innovative treatment modalities, including CAR T-cell therapy, TIL (Tumor-Infiltrating Lymphocyte) therapy, and NK (Natural Killer) cell therapy.

Hau's expertise lies in cancer cell biology, where she has made significant contributions to understanding the complex interactions between immune cells and tumors.

Her research aims to enhance the efficacy of immunotherapies by manipulating the tumor microenvironment and exploring novel ways to activate and direct immune responses against cancer cells.

Throughout her career, Hau has collaborated with leading professors and researchers in the field of cancer treatment, both in the United States and China.

These international experiences have broadened her perspective and contributed to her innovative approach to cancer therapy development.

Hau's work is particularly focused on addressing the challenges of treating advanced and metastatic cancers. She has been involved in clinical trials evaluating the safety and efficacy of various immunotherapy approaches, including the promising Gamma Delta T cell therapy.

• Susan Hau

  https://cancerfax.com/author/susan/

  Gene Therapy for Gaucher’s Disease in China
• Susan Hau

  https://cancerfax.com/author/susan/

  Gene Therapy for Wilson’s Disease in China
• Susan Hau

  https://cancerfax.com/author/susan/

  Gene Therapy for Phenylketonuria in China
• Susan Hau

  https://cancerfax.com/author/susan/

  Best Cancer Hospitals in China 2025