The US Food and Drug Administration (FDA) approved ivosidenib (Tibsovo®) for the treatment of patients with previously treated isocitrate dehydrogenase 1 (IDH1)-mutated cholangiocarcinoma.
“We are proud to bring to patients the first and only targeted therapy for previously treated IDH1-mutated cholangiocarcinoma,” David K. Lee, chief executive officer of Servier Pharmaceuticals, the manufacturer of ivosidenib, said in a press release.
Cholangiocarcinoma is a rare type of cancer that affects the bile ducts. Approximately 8000 people are diagnosed with the disease each year in the US. Around 20% of them have an IDH1 mutation. Thus far, there have been no approved targeted therapies for cholangiocarcinoma patients with an IDH1 mutation.
Ivosidenib is an IDH1 inhibitor. It was already approved as a monotherapy for the treatment of adult patients with acute myeloid leukemia (AML) with an IDH1 mutation.
Read more about cholangiocarcinoma treatment
The approval of the treatment in patients with cholangiocarcinoma is based on the results of a multicenter, randomized, double-blind, placebo-controlled, phase 3 clinical trial called ClarIDHy that investigated patient response to treatment.
The results showed that overall survival was better in patients receiving ivosidenib than in those receiving placebo, although the difference was not statistically significant. The median overall survival rates were 10.3 months for ivosidenib and 7.5 months for placebo. The most common adverse effects associated with the treatment were fatigue, nausea, abdominal pain, diarrhea, coughing, reduced appetite, ascites, vomiting, anemia, and rash.
Rachna T. Shroff, MD, associate professor of medicine at the University of Arizona and chief of gastrointestinal medical oncology at the University of Arizona Cancer Center, stated, “In addition to an acceptable safety profile, Tibsovo demonstrated an impressive, significant benefit in progression-free survival, underscoring its importance as a new option for patients battling this aggressive cancer.”
The recommended dosage of ivosidenib for previously treated cholangiocarcinoma patients with mutated IDH1 is 500 mg orally once a day with or without food until disease progression or unacceptable toxicity occurs.
Servier announces FDA approval of TIBSOVO® (ivosidenib tablets) in IDH1-mutated cholangiocarcinoma. News release. Servier Pharmaceuticals; August 25, 2021.
Study of AG-120 in previously treated advanced cholangiocarcinoma with IDH1 mutations (ClarIDHy) (ClarIDHy). ClinicalTrials.gov. December 12, 2016. Updated May 21, 2021. Accessed August 26, 2021.