Express for the treatment of cholangiocarcinoma, innovative FGFR inhibitor has been granted priority review by the FDA

▎WuXi AppTec Content Team Editor Today, Japan's Taiho Pharmaceutical and its subsidiary Taiho Oncology announced that the US FDA has accepted a New Drug Application (NDA) for futibatinib for the treatment of localized patients with FGFR2 gene rearrangements (including gene fusions).
Treatment-experienced patients with advanced or metastatic cholangiocarcinoma
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Futibatinib is a potent and selective irreversible oral small molecule inhibitor of FGFR1, 2, 3, and 4
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The FDA also granted the application priority review status and is expected to respond by September 30 this year
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Cholangiocarcinoma includes intrahepatic and extrahepatic cholangiocarcinoma, and it usually occurs in the elderly
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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
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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
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The 5-year survival rate for intrahepatic cholangiocarcinoma is only 9%, and the incidence is higher in Asian populations
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Futibatinib inhibits FGFR-mediated signaling by irreversibly covalently binding to the ATP-binding "pocket" of FGFR1-4, thereby reducing the proliferation of tumor cells harboring FGFR1-4 genetic variants
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It has been granted Breakthrough Therapy Designation and Orphan Drug Designation by the FDA for the treatment of cholangiocarcinoma
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▲ Schematic diagram of the mechanism of action of futibatinib (Image source: Reference [2]) This application is based on data from a pivotal Phase 2b clinical trial in which 103 patients were treated with futibatinib
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The primary endpoint of the clinical trial was objective response rate (ORR), futibatinib had an ORR of 41.
7%, and the key secondary endpoint was duration of response (DOR), with a median DOR of 9.
7 months for futibatinib and a DOR of 72% of patients who achieved a response more than 6 months
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"This is an important step towards our goal of bringing futibatinib to patients in need of new treatment options,
"
said Mr.
Teruhiro Utsugi, Senior Managing Director of Taiho Pharmaceutical
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Reference: [1] US Food and Drug Administration (FDA) Accepts for Priority Review Taiho Oncology's New Drug Application for Futibatinib for Cholangiocarcinoma.
Retrieved March 30, 2022, from https:// -food-and-drug-administration-fda-accepts-for-priority-review-taiho-oncologys-new-drug-application-for-futibatinib-for-cholangiocarcinoma-301513278.
html[2] Sootome et al.
, (2020 ).
Futibatinib Is a Novel Irreversible FGFR 1–4 Inhibitor That Shows Selective Antitumor Activity against FGFR-Deregulated Tumors.
Cancer Research, DOI: 10.
1158/0008-5472.
CAN-19-2568 Advances in Biomedical Health Research
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This article is also not a treatment plan recommendation
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For guidance on treatment options, please visit a regular hospital
.