FDA Grants Accelerated Approval to Futibatinib for Cholangiocarcinoma

The recent FDA approval for futibatinib was based on a clinical study that was opened in our clinic at Mary Crowley Cancer Research.

The drug tested was designed to inhibit an abnormal growth signal that occurs in some cases of a cancer called intrahepatic cholangiocarcinoma. This cancer is a tumor of the bile ducts within the liver. The tumor is difficult to detect in its early stages. Advanced or metastatic tumors of this type are treated with chemotherapy but usually become resistant.

A gene fusion involving the FGFR2 gene is present in about 15% of these tumors. FGFR2 stands for fibroblast growth factor receptor 2. There are several fibroblast growth factors and there are 4 fibroblast growth factor receptors. The normal FGF receptor gives a growth signal in cells that have the receptor when receptor binds to FGF detected on the surface of the cell. The mutant receptor produced by the gene fusion in cholangiocarcinoma cancer cells sends the growth signal regardless of whether FGF is present. Therefore, blocking signaling by the mutant receptor could stop growth of these tumors.

This new drug was used in study #17-23 and in study #20-20 in our clinic and was open to patients with tumors found by tumor DNA sequencing to have a gene fusion involving FGFR2. Most patients with tumors having this abnormality have intrahepatic cholangiocarcinoma, but abnormalities of FGFR2 are found in some cases of breast cancer, endometrial cancer, gastric cancer, as well as rare cases in other types of cancer.

In this study patients received a daily dose of futibatinib in tablet form. The response rate for patients with intrahepatic cholangiocarcinoma, as measured by decrease in tumor size on follow-up CT scans, was 42%. The average duration of response was 9.7 months.

The approval by the FDA was base based on results including study #17-23. The new drug is made by Taiho Oncology with the brand name Lytgobi. Further studies are in progress to compare this drug against chemotherapy for intrahepatic cholangiocarcinoma and to test for activity in other cancers with abnormalities of the FGFR2 gene.

This approval is the 19th drug approved by the FDA using results in part from studies conducted in our clinic.