Regulatory approval

Published by the Food and Drug Administration.

The U.S. Food and Drug Administration granted approval to ivosidenib for the treatment adult patients with locally advanced or metastatic cholangiocarcinoma who have been previously treated with a susceptible IDH1 mutation, as detected by an FDA-approved test. This indication is based on Study AG120-C-005 (NCT02989857), a randomized, multicenter, double-blind, placebo-controlled clinical trial consisting of 185 adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 mutation. The product label notes that, across both arms of the trial, patients primarily had R132 variants: R132C (70% of patients), R132L (15%), R132G (12%), R132H (1.1%), and R132S (1.6%).

This is written in the approval document as:

TIBSOVO is an isocitrate dehydrogenase-1 (IDH1) inhibitor indicated for patients with a susceptible IDH1 mutation as detected by an FDA-approved test with for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma who have been previously treated.

Citation

Agios Pharmaceuticals, Inc. Tibsovo (ivosidenib) [package insert]. U.S. Food and Drug Administration website. https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/211192s011lbl.pdf. Revised October 2023. Accessed October 30, 2024.

Therapeutic response

Precision oncology relationships for therapeutic response derived from this regulatory approval.

Type	Biomarker(s)	Cancer type	Therapy(ies)
Sensitivity (+)	IDH1 p.R132C	Cholangiocarcinoma	Ivosidenib
Sensitivity (+)	IDH1 p.R132G	Cholangiocarcinoma	Ivosidenib
Sensitivity (+)	IDH1 p.R132H	Cholangiocarcinoma	Ivosidenib
Sensitivity (+)	IDH1 p.R132L	Cholangiocarcinoma	Ivosidenib
Sensitivity (+)	IDH1 p.R132S	Cholangiocarcinoma	Ivosidenib