Cholangiocarcinoma: Ivosidenib FDA Approval for IDH1-Mutant Disease

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

FDA Approval: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-ivosidenib-advanced-or-metastatic-cholangiocarcinoma

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