Agios Submits sNDA for TIBSOVO for IDH1-Mutant Cholangiocarcinoma

Agios Pharmaceuticals has submitted a Supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for TIBSOVO® (ivosidenib tablets) as a potential treatment for patients with previously treated isocitrate dehydrogenase 1 (IDH1) mutated cholangiocarcinoma. Agios has requested priority review for the application, which, if granted, could result in a six-month review process.

“Cholangiocarcinoma is a rare, aggressive cancer with limited effective therapies, and patients are in desperate need of new treatment options – particularly those who experience disease progression after chemotherapy,” said Chris Bowden, M.D., chief medical officer at Agios. “We are proud of the work we have done on behalf of these patients and look forward to working closely with the FDA during the review of the first oral therapy targeting an IDH1 mutation for patients with previously treated IDH1-mutated cholangiocarcinoma.”

The sNDA submission is supported by data from the ClarIDHy study, the first and only randomized Phase 3 trial for previously treated IDH1-mutated cholangiocarcinoma. Data from the study were previously presented at the European Society for Medical Oncology Congress (ESMO), held in September 2019 in Barcelona, Spain, and published in The Lancet Oncology on May 13, 2020. A final analysis of the data was featured in an oral presentation at the American Society of Clinical Oncology Gastrointestinal Cancers Symposium (ASCO-GI) on January 17, 2021.

Cholangiocarcinoma is a rare, aggressive cancer of the bile ducts within and outside of the liver. IDH1 mutations occur in approximately 13% of cholangiocarcinoma cases and are not associated with prognosis. There are no approved systemic therapies for IDH1-mutated cholangiocarcinoma and limited chemotherapy options are available in the advanced setting. Gemcitabine-based chemotherapy is often recommended for newly diagnosed advanced or metastatic disease.