Express|Innovative precision therapy for the treatment of cholangiocarcinoma received accelerated approval from the FDA

▎WuXi AppTec Content Team Editor

Today, Taiho Pharmaceutical and its subsidiary Taiho Oncology announced that the U.S. FDA has accelerated the approval of Lytgobi (futibatinib) for the treatment of patients with FGFR2 gene fusion or other serious diseases. Treatment-experienced adult patients with unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma. This therapy achieved a 42% objective response rate and a 9.7-month median duration of response in pivotal clinical trials.

cholangiocarcinoma is an aggressive cancer that develops in the bile ducts. It includes both intrahepatic and extrahepatic types. About 20% of cholangiocarcinoma patients are intrahepatic cholangiocarcinoma, and more than 10-16% of these patients carry FGFR2 gene rearrangements. At present, the main treatment for cholangiocarcinoma is surgical resection. However, there are no obvious symptoms in the early stage of cholangiocarcinoma, and most patients have lost the opportunity for surgery when they are diagnosed. Locally advanced and metastatic cholangiocarcinoma cannot be completely removed by surgery. The current standard treatment options are mainly chemotherapy, radiotherapy, and liver transplantation, and the prognosis of patients is poor. The 5-year survival rate for intrahepatic cholangiocarcinoma is only 9%, and the incidence is higher in Asian populations.

Futibatinib inhibits FGFR-mediated signaling by irreversibly covalently binding to the ATP-binding “pocket” of FGFR1-4, thereby reducing tumor cells harboring FGFR1-4 mutations proliferation. It was FDA approved

Breakthrough Therapy Designation

, Orphan Drug Designation and Priority Review Designation for the treatment of cholangiocarcinoma.

This approval is based on data from a pivotal Phase 2b clinical trial showing futibatinib achieved an objective response rate of 42% with a median duration of response of 9.7 months, 72% of patients had remissions lasting more than 6 months.

“Lytgobi provides an effective, well-tolerated oral therapy for patients with intrahepatic biliary tract cancer.” said Mr. Tim Whitten, President and CEO of Taiho Oncology. “This approval This is an important milestone for patients and offers hope for improved outcomes.”