Cholangiocarcinoma Foundation commends FDA approval of new therapy for cholangiocarcinoma patients

CCF provided patient insights into Servier’s development of TIBSOVO® as a treatment for individuals with IDH1-mutated cholangiocarcinoma

SALT LAKE CITY, UT – The Cholangiocarcinoma Foundation (CCF) today applauds the U.S. Food and Drug Administration (FDA) approval of TIBSOVO® (ivosidenib tablets), a new therapy for cholangiocarcinoma patients developed by Servier Pharmaceuticals. As part of its work to ensure all drug development incorporates the views of patients, the Foundation collaborated with Servier by providing patient insights. CCF gave vital input on the design of the clinical trial, which evaluated the new drug for use in patients with IDH1 cholangiocarcinoma (CCA).

Final FDA approval of TIBSOVO® was granted after a Priority Review, which accelerated the review of the company's supplemental New Drug Application (sNDA) from 10 months to less than 6 months from the day of filing acceptance. Priority Review is typically given to drugs that may offer major advances in treatment or may provide a treatment where no adequate therapy exists.

The sNDA acceptance was supported by data from Agios’ global ClarIDHy study, the first and only randomized Phase 3 trial for previously treated IDH1-mutated cholangiocarcinoma. An encore presentation of the data was presented at the American Society of Clinical Oncology (ASCO) Annual Meeting on June 4-8, 2021.

“We are grateful to Servier for its commitment to this patient population, to the FDA for its commitment to timely review, and to the patients and clinicians who committed themselves to the clinical trials that delivered the necessary results for approval,” said Stacie Lindsey, CCF founder and CEO. “This new treatment will positively impact the lives of patients diagnosed with IDH1-mutated cholangiocarcinoma and will advance our progress for the whole cholangiocarcinoma community.”

The Foundation’s initial engagement in trial development was predicated on the significant unmet need of this patient population. Limited chemotherapy options are available in the advanced setting.

“We are proud and honored to have the opportunity to deliver a first in class targeted systemic treatment to patients with previously treated IDH1-mutated cholangiocarcinoma,” said Susan Pandya MD, Vice President and Global Head of Cancer Metabolism at Servier Pharmaceuticals. “We remain steadfast in our commitment to addressing the unmet needs of the cholangiocarcinoma community, and are grateful to the courageous patients who helped pave the way towards progress in this disease.”

The Foundation is the leading global resource for research, education, and public awareness for CCA patients and is celebrating its 15th anniversary this year. More information is available here https://bit.ly/3A6WPft.