Cholangiocarcinoma: Ivosidenib FDA Approval for IDH1-Mutant Disease
Medical Supporter — Information Notice
This article is a summary of international medical information and is not medical advice; it cannot replace the diagnosis or treatment plan of your attending physician. The medical technologies, drug information and clinical data presented here are compiled from public literature and official statements of major Japanese medical institutions; the applicability and outcome of any therapy vary with each patient and must be assessed individually by a qualified physician.
FDA Approval Announcement
- September 2, 2021
On August 25, 2021, the FDA approved ivosidenib (Tibsovo) for previously treated, locally advanced or metastatic IDH1-mutant cholangiocarcinoma. FDA also approved Oncomine Dx Target Test as companion diagnostic for IDH1 mutation detection.
AG120-C-005 Trial Design
Patient Population: IDH1-mutant previously treated locally advanced/metastatic cholangiocarcinoma (n=185)
Treatment Arms (2:1 randomization):
- Ivosidenib 500 mg once daily oral (n=124)
- Placebo (n=61)
Primary Endpoint: Progression-free survival
Treatment Duration: Until disease progression or unmanageable toxicity
Key Efficacy Results
Progression-Free Survival: Significant improvement with ivosidenib versus placebo
Overall Survival: No significant difference between arms
Crossover: ~70% of placebo patients crossed over to ivosidenib after documented disease progression
Safety Profile
Most Common Adverse Events (≥15%):
- Fatigue
- Nausea
- Abdominal pain
- Diarrhea
- Cough
- Anorexia
- Ascites
- Vomiting
- Anemia
- Rash
Dosing and Administration
Recommended Dose: Ivosidenib 500 mg orally once daily (with or without food)
Treatment Duration: Until disease progression or unmanageable toxicity
Clinical Significance
Ivosidenib provides oral targeted therapy option for IDH1-mutant cholangiocarcinoma, expanding treatment paradigm for this genetically defined subset.
Data Source
Medical Supporter was formerly certified as an international medical visa guarantor by Japan's Ministry of Foreign Affairs and the Ministry of Economy, Trade and Industry (B-066).
Considering medical care in Japan? Need information and support?
We help you organize the information needed for medical travel to Japan, liaise with Japanese medical institutions, and arrange a second-opinion consultation.The first consultation is free; an advisor will help you clarify the next steps.
Figure 1
Figure 2
Figure 3
Figure 4