FDA Approves Tibsovo for Rare, Aggressive Cancer

The FDA has approved Servier Pharmaceuticals’ Tibsovo (ivosidenib) to treat adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with an IDH1. TIBSOVO is the first and only targeted therapy approved for patients with previously treated IDH1-mutated cholangiocarcinoma, a cancer of the bile ducts within and outside of the liver.

An estimated 8,000 people in the United States are diagnosed with cholangiocarcinoma each year. But the actual number of these cases is likely higher, as this cancer can be hard to diagnose, and may be misclassified as other types of cancer.

Tibsovo is also approved as monotherapy for the treatment of adults with IDH1-mutated relapsed or refractory acute myeloid leukemia (AML) and for adults with newly diagnosed IDH1-mutated AML who are ≥75 years old. The supplemental NDA for the cholangiocarcinoma indication had received priority review.

The approval was based on the ClarIDHy clinical trial, which found overall survival (OS) favoring patients randomized to Tibsovo compared with those randomized to placebo. The safety profile observed in the study was consistent with previously published data.

At the same time, the FDA approved Thermo Fisher Scientific’s Oncomine Dx Target Test as a companion diagnostic (CDx) to identify patients with IDH1-mutated cholangiocarcinoma who may be candidates for Tibsovo.

The Oncomine Dx Target Test is a next-generation sequencing (NGS) based test that delivers reproducible results in the IDH1 gene clinically associated with CCA. The FDA first approved the test as a CDx in 2017, and it is now approved for four targeted therapies for non-small cell lung cancer and one targeted therapy for CCA in the United States. The test is currently approved and reimbursed by government and commercial insurers in more than 15 countries.

Stacie LIndsey

“Before the approval of Tibsovo, there were no approved targeted therapies available to cholangiocarcinoma patients harboring the IDH1 mutation, and limited chemotherapy options available to patients with advanced disease,” Stacie Lindsey, founder and CEO, Cholangiocarcinoma Foundation, said in a statement. “This approval brings new hope to the cholangiocarcinoma community, and we are excited that this much-needed new therapeutic option is being made available to patients.”