CCF Celebrates FDA’s Filing Acceptance and Priority Review of TIBSOVO® for the Treatment of IDH1-mutations in CCA

On behalf of patients and families impacted by cholangiocarcinoma (CCA), the Cholangiocarcinoma Foundation (CCF) today hailed progress in the development journey for TIBSOVO® (ivosidenib tablets) a targeted therapy for IDH1-mutated CCA patients, as Servier Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA) for TIBSOVO (ivosidenib tablets) as a potential treatment for patients with previously treated IDH1-mutated cholangiocarcinoma.

The sNDA was granted Priority Review, which accelerates the review time from 10 months to a goal of 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 is supported by the positive primary analysis of progression-free survival and the recent encouraging overall survival (OS) analysis from Agios' global Phase 3 ClarIDHy trial of TIBSOVO® in previously treated CCA patients with an isocitrate dehydrogenase 1 (IDH1) mutation.

CCF has been involved in the global effort to advance this approach for treating the IDH1-positive subset of CCA patients – which accounts for approximately 20% of Intrahepatic CCA cases -- since the beginning of the Servier Pharmaceuticals development program, providing patient insights and input on the design of the trial. CCF 's initial engagement was predicated on the significant unmet need of this patient population, as there are no approved systemic therapies. Limited chemotherapy options are available in the advanced setting. Gemcitabine-based chemotherapy is often recommended for newly diagnosed advanced or metastatic disease.

"This is an important milestone for this community and specifically for patients with an IDH1 mutation. We want to acknowledge all who participated in this trial, and thank them for playing such a critical role. This approval shows that TIBSOVO® has the potential to lengthen time to disease progression for these patients and provides hope that they will have access to a viable treatment option" said Stacie Lindsey, Founder and CEO, CCF.

"Currently, there are no approved systemic therapies for IDH1-mutated cholangiocarcinoma and limited chemotherapy options are available for patients with advanced disease," said Susan Pandya, M.D., Vice President, Clinical Development, Head of Cancer Metabolism Global Development, Servier Pharmaceuticals. "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."

"Facing a stage 4 cholangiocarcinoma diagnosis, my future was unclear. Tibsovo gave me a future, it gave my life back! For the last two and half years, I have lived a normal life, without tumor progression, drug or cancer side effects. I feel like I hit the cancer lotto with Agios' TIBSOVO®," said Kathy Barboza, cholangiocarcinoma patient and AG 120 trial participant.