Express | Disease control rate exceeds 80%, innovative FGFR inhibitors are recognized as breakthrough therapy by FDA

▎The content team editor of WuXi AppTec.
Today, Taiho Pharmaceutical and its subsidiary Taiho Oncology announced that the US FDA has granted the irreversible FGFR oral inhibitor futibatinib (TAS-120) breakthrough therapy designation for the treatment of locally advanced treatment.
Or patients with metastatic cholangiocarcinoma (cholangiocarcinoma).

These patients carry the FGFR2 gene rearrangement or fusion.

Breakthrough therapy designation aims to accelerate the development and review process of innovative therapies that treat severe or life-threatening treatments.

Cholangiocarcinoma includes intrahepatic cholangiocarcinoma and extrahepatic cholangiocarcinoma, and it usually occurs in the elderly.

At present, the main treatment for cholangiocarcinoma is surgical resection, but 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 chemotherapy, radiotherapy, and liver transplantation.
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.

Last year, the FDA accelerated the approval of Pemazyre (pemigatinib), an FGFR2 inhibitor developed by Incyte, to treat adult patients with FGFR2 gene fusion or other rearrangements that have been treated for advanced cholangiocarcinoma.

Brings the first targeted therapy for the treatment of adult patients with advanced cholangiocarcinoma.

Futibatinib is an oral, selective, and irreversible FGFR1-4 small molecule inhibitor.

It irreversibly covalently binds to the ATP binding "pocket" of FGFR1-4 to inhibit FGFR-mediated signal transduction, thereby reducing the proliferation of tumor cells carrying FGFR1-4 gene mutations.

It has been granted orphan drug designation by the FDA in 2018 for the treatment of cholangiocarcinoma.

▲Schematic diagram of the mechanism of action of Futibatinib (picture source: reference [3]) The grant of this breakthrough therapy designation is based on the results of a phase 2 clinical trial called FOENIX-CCA2.

An analysis of interim data published at the European Society of Medical Oncology Asia Conference (ESMO Asia) last year showed that among 67 patients with intrahepatic cholangiocarcinoma with FGFR2 gene fusion or rearrangement after a follow-up period of more than 6 months, futibatinib reached 37.
3% Objective response rate (ORR), disease control rate of 82.
1%, median duration of response reached 8.
3 months, and median progression-free survival reached 7.
2 months.

▲ Interim analysis results of Futibatinib in phase 2 clinical trials (picture source: Reference [2]) Patients in different subgroups all showed objective remission.
It is worth mentioning that in the subgroup of patients over 65 years of age, ORR Reached 57.
1%.

The latest results of this clinical trial will be announced at the AACR annual meeting in April this year.

In China, many biopharmaceutical companies, including Nuocheng Jianhua, Cinda Bio, CStone Pharmaceuticals, and Zai Lab, are also developing different types of FGFR inhibitors for the treatment of advanced cholangiocarcinoma, hepatocellular carcinoma, and gastric cancer.
And other types of cancer patients.

For details, please refer to: Nuocheng Jianhua, Cinda Biological, CStone Pharmaceuticals, etc.
are all developing, can FGFR become a new star of "unlimited cancer" therapy? Reference: [1] FDA Grants Breakthrough Therapy Designation for Taiho Oncology's Futibatinib for Treatment of Advanced Cholangiocarcinoma.
Retrieved April 1, 2021, from designation-for-taiho-oncologys-futibatinib-for-treatment-of-advanced-cholangiocarcinoma-301260615.
html[2] FUTABATINIB FOR PATIENTS WITH INTRAHEPATIC CHOLANGIOCARCINOMA AND FGFR2 FUSIONS OR REARRANGEMENTS.
Retrieved:// April 1, 2021, from https .
esmo.
org/oncology-news/futabatinib-for-patients-with-intrahepatic-cholangiocarcinoma-and-fgfr2-fusions-or-rearrangements[3] 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.

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