EU Commission Approval of Tibsovo® (Ivosidenib) in IDH1-mutated Cholangiocarcinoma

July 17, 2023

The European Commission approved the use of Tibsovo® (Ivosidenib) as monotherapy for the treatment of locally advanced or metastatic cholangiocarcinoma with an isocitrate dehydrogenase-1 (IDH1) R132 mutation. Adult patients who were previously treated by at least one prior line of systemic therapy are eligible for this novel therapeutic option1.

Cholangiocarcinoma starts in the bile ducts (tubes that carry bile to the gallbladder). It is a rare and aggressive form of cancer, which affects 1–3 in 100,000 people in Europe, with approximately 10,000 new cases each year2. To date, only surgery has been shown to provide a cure, but this is only possible for a small number of patients, and the risk of relapse is high. Chemotherapy and immunotherapy are the standard treatment for patients who are not eligible for surgery, and whose disease has progressed after surgery3

The European Commission’s approval is based on data from the ClarIDHy trial, a randomised Phase 3 trial for previously treated IDH1-mutated cholangiocarcinoma4. Results from the ClarIDHy study demonstrated a statistically significant improvement in f progression-free survival (PFS) by an independent review committee (HR 0.37; 95% CI [0.25, 0.54], p<0.001). The median PFS (95% CI) for Tibsovo® and placebo was 2.7 (1.6, 4.2) and 1.4 (1.4, 1.6) months, respectively. Out of the patients randomized to Tibsovo®, 32% and 22% remained free of progression or death at 6 and 12 months, respectively, versus none on the placebo arm. The most common adverse reactions were fatigue, nausea, abdominal pain, diarrhoea, decreased appetite, ascites, vomiting, anaemia, and rash.

Tibsovo® is the first and only IDH1 inhibitor approved in Europe. It provides a significant benefit to patients by over-available therapies for cholangiocarcinoma4.


 Tibsovo | European Medicines Agency.

2  Valle, J. W. et al. Biliary cancer: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol 27, v28–v37 (2016).

3  Oliveira, I. S. et al. Cholangiocarcinoma: classification, diagnosis, staging, imaging features, and management. Abdom Radiol (NY) 42, 1637–1649 (2017).

4  Abou-Alfa, G. K. et al. Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study. Lancet Oncol 21, 796–807 (2020).

Lili Gundelach

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