It's been a month of progress in the world of Cholangiocarcinoma as the FDA grants priority review to an IDH1 inhibitor Ivosidenib, weeks after breakthrough designation for Futibatinb.

In April the FDA granted Breakthrough Therapy Designation for futibatinib in intrahepatic cholangiocarcinoma tumours positive for FGFR2 fusions or rearrangements. This month marks another promising breakthrough in cholangiocarcinoma, with the FDA granting priority review to ivosidenib for patients with previously treated, IDH1-mutant cholangiocarcinoma, and we are delighted to see our experts are in the driving seat.

The Rare Initiative’s Dr. Lipika Goyal has been working with Servier to develop ivosidenib, a drug that inhibits mutated IDH1 protein. Susan Pandya, MD, vice president of Clinical Development and head of Cancer Metabolism Global Development at Servier Pharmaceuticals, stated in a press release, “Currently, there are no approved systemic therapies for IDH1-mutated cholangiocarcinoma and limited chemotherapy options are available for patients with advanced disease.” But IDH1 gene mutations are found in some advanced cholangiocarcinoma tumors, so ivosidenib is a promising targeted therapy for patients with limited options and little hope.

This drug, which is sold under the brand name Tibsovo, is already used to treat IDH1-mutant leukemia. But the recent phase 3 ClarIDHy trial studied the benefits of ivosidenib in IDH1-mutant cholangiocarcinoma and showed a 63% reduction in the risk of disease progression or death in comparison to the placebo.

With an outcome like this, it comes as no surprise that the FDA granted priority review to the supplemental new drug application for ivosidenib for patients with previously treated, IDH1-mutant cholangiocarcinoma. The priority review means ivosidenib will be reviewed in 6 months, instead of 10 months with a standard review  – and with liver cancer and cholangiocarcinoma predicted to be the third leading cause of cancer death by 2040, this expedited timeline will bring real hope to those who don’t have time to waste.

Dr. Susan Pandya sums up the importance of this breakthrough, “The FDA’s priority review is a major milestone for patients. I’d like to acknowledge and thank all the patients, their families, and the investigators and research teams who took part in the ClarIDHy study.”

It was almost exactly two years ago, in May 2019, that the FDA approved ivosidenib for use in patients with IDH1-mutated acute myeloid leukemia. In this instance, ivosidenib was approved for use specifically in those whose disease is newly diagnosed, who are at least 75 years of age, who can’t use certain chemotherapy treatments due to other health problems, and who have relapsed or refractory acute myeloid leukemia.

The possibility of Ivosidenib being approved a second time for use in IDH1-mutant cholangiocarcinoma proves that targeted therapies are the future of cancer treatment – and that discovery in any cancer could mean progress for all cancers.

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