August 25, 2021 – Boston
Servier Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has approved Ivosidenib for the treatment of adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma harboring an IDH1 mutation, as detected by an FDA-approved test. Ivosidenib is the first and only targeted therapy approved for patients with previously treated, IDH1-mutated cholangiocarcinoma.
The Supplemental New Drug Application (sNDA) for Ivosidenib was granted Priority Review designation, which accelerates the review timeline and is typically granted to drugs that may offer significant therapeutic advances or provide a treatment where adequate therapies do not currently exist. David K. Lee, CEO of Servier Pharmaceuticals, stated: "Servier has long been committed to exploring the immense potential of inhibiting mutated IDH enzymes as a novel therapeutic approach for cancers with high unmet needs, including cholangiocarcinoma. We are proud to bring patients the first and only targeted therapy for previously treated cholangiocarcinoma with an IDH1 mutation. We are grateful to the patients, caregivers, investigators, and study teams whose participation in the ClarIDHy clinical trial made this achievement possible."
Dr. Rachna T. Shroff, Associate Professor of Medicine at the University of Arizona and Chief of Gastrointestinal Medical Oncology at the University of Arizona Cancer Center, commented: "Patients with IDH1-mutated cholangiocarcinoma—particularly those whose disease has progressed following chemotherapy—are in urgent need of new treatment options. In addition to an acceptable safety profile, Tibsovo demonstrated impressive and significant benefits in terms of progression-free survival, underscoring its importance as a new option for patients battling this aggressive cancer."
Stacie Lindsey, Founder and CEO of the Cholangiocarcinoma Foundation, said: "Prior to today's approval of Ivosidenib, there were no approved targeted therapies available for patients with IDH1-mutated cholangiocarcinoma, and chemotherapy options for those with advanced disease were extremely limited. This approval brings new hope to the cholangiocarcinoma community, and we are delighted that this much-needed new treatment option is now available to patients."
Ivosidenib: Dosage and Administration
The recommended dosage of Ivosidenib for previously treated cholangiocarcinoma with an IDH1 mutation is 500 mg, taken orally once daily, with or without food, until disease progression or unacceptable toxicity occurs.
