The FDA recently granted Breakthrough Therapy Designation for futibatinib in cholangiocarcinoma tumors positive for FGFR2 gene mutations.

With liver cancer and cholangiocarcinoma (CCA) predicted to become the third leading cause of cancer death in both men and women by 2040 , this is a breakthrough worth shouting about. So let’s break it down.

Intrahepatic CCA is a rare but aggressive tumour of the bile ducts within the liver. Patients face a poor prognosis due to late stage diagnosis, the chemoresistance of the disease, and limited treatment options. However, the discovery of actionable targets in 40-50% of intrahepatic CCAs is shifting the treatment paradigm in this disease, with multiple targeted therapies now in clinical trials. That’s where futibatinib comes in…

One promising actionable target is the fibroblast growth factor receptor 2 (FGFR2) gene fusion, a genetic alteration that occurs in 10-15% of patients with intrahepatic CCA. Futibatinib is an irreversible FGFR inhibitor which was evaluated in the FOENIX-CCA2 study, a global phase II trial in 103 patients with intrahepatic CCA that harbor FGFR2 gene fusions or rearrangements.

During the trial, futibatinib showed an encouraging 42% response rate in patients who have previously been treated with gemcitabine and platinum-based chemotherapy. That’s potentially life-changing for 42% of patients with intrahepatic CCA.

Rare Initiative Gamechanger Dr. Lipika Goyal was the principal investigator of the FOENIX-CCA2 study. Listen to her discuss the results of this phase II trial and the clinical benefit of futibatinib here. Or if you’re suddenly regretting not paying more attention in science class, here’s what you need to know:

  • The responses occurred early and were durable, with a median time to response of 2.5 months and a median duration of response of 9.7 months.
  • The disease control rate was 82.5%, with the majority of patients with stable disease experiencing tumor shrinkage.
  • The safety profile of the drug and previously reported quality of life data indicated that the drug was well-tolerated.
Alongside Taiho Oncology Inc., Massachusetts General Hospital has been one of the leaders in the development of this drug, from the first in-human phase I study in all solid tumors, to the phase II global study. On April 11 2021, encouraging new data from FOENIX-CCA2 was presented on futibatinib at the Clinical Trial Plenary Session of the American Association of Cancer Research Annual Conference, one of the largest scientific oncology conferences of the year.


The results speak for themselves, so on April 1 2021, the FDA granted futibatinib Breakthrough Therapy Designation for advanced cholangiocarcinoma. This means the drug will get expedited review, bringing real hope to those with limited treatment options. In a press release, Teruhiro Utsugi, PhD, the senior managing director at Taiho, said:

"We will continue to advance our research and development efforts to deliver futibatinib, discovered in our research center, as one of the agents which may benefit cholangiocarcinoma patients around the world waiting for new treatment options.”

The importance of developing targeted therapies in clinical trials cannot be understated. This breakthrough with futibatinib is a glimpse of the future of cancer treatment, and an example of what our funding can achieve. But this is just the beginning.

Learn about the research we are funding and make a donation to help more game-changing drugs gain FDA approval.