培美替尼是首个获批的口服肝内胆管癌靶向药

Is the first approved oral targeted drug for intrahepatic cholangiocarcinoma

Cholangiocarcinoma is a malignant tumor originating from bile duct epithelial cells. It can be divided into two categories: intrahepatic cholangiocarcinoma and extrahepatic cholangiocarcinoma according to the location of occurrence. In recent years, the incidence of cholangiocarcinoma has been increasing year by year. Surgery is the only treatment with curative potential, but only a small number of early-stage patients have the opportunity for surgery at the time of diagnosis. The recurrence rate of patients with radical resection is still high, and the current systemic treatments for unresectable or advanced cholangiocarcinoma have poor efficacy. Standard treatment (chemotherapy regimen of cisplatin combined with gemcitabine) is recommended as the first-line treatment for advanced cholangiocarcinoma. The objective response rate (ORR) is 15~26%, and drug resistance often occurs, and the overall survival is less than 1 year. The launch of pemetinib has changed this situation.

In April 2020, the U.S. Food and Drug Administration accelerated the approval of the intrahepatic cholangiocarcinoma targeting drug pemetinib, which is suitable for patients with previously treated locally advanced or metastatic cholangiocarcinoma who have FGFR2 gene fusion or rearrangement. FGFR2 gene fusion or rearrangement is almost only seen in intrahepatic cholangiocarcinoma, so pemetinib is an intrahepatic cholangiocarcinoma-targeted drug. It is also the first intrahepatic cholangiocarcinoma-targeted drug.

According to the results of the Phase II clinical trial of pemetinib, this pemetinib study divided all cholangiocarcinoma patients into three groups: 107 patients with cholangiocarcinoma FGFR2 gene fusion or rearrangement, 20 patients with other FGF/FGFR gene mutations, and 18 patients without FGF/FGFR gene mutations. After a period of clinical use of the new drug pemetinib for intrahepatic cholangiocarcinoma once daily, in patients with FGFR2 gene fusion or rearrangement, the objective response rate of pemetinib reached 35.5%, the disease control rate was 88.2%, the median progression-free survival was 6.9 months, and the median overall survival was 21.1 months.

From this, it can be seen that the new drug pemetinib for cholangiocarcinoma is more suitable for patients with cholangiocarcinoma with FGFR2 gene rearrangement or fusion, and pemetinib has good clinical effects on patients with cholangiocarcinoma.

Note: The above information comes from the Internet and is compiled and edited by Medical Companion Travel (please correct me if there are any errors or omissions). It is only to provide information on the latest drugs on the market in the world and help Chinese patients understand the latest international new drug trends. It is only for internal discussion among medical staff and does not serve as any basis for medication. For specific medication guidelines, please consult the attending physician.

Recommended related articles: