What are the results of the clinical trial of the cholangiocarcinoma-targeted drug pemetinib?

Pemetinib (Pemetinib ), developed by Incyte Corporation, is a targeted drug targeting fibroblast growth factor receptor (FGFR) mutations. It is mainly used to treat patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 gene fusions or rearrangements. Pemetinib effectively blocks the growth and spread of cancer cells by inhibiting the FGFR signaling pathway, providing a new treatment option for these patients.

Clinical trial results of pemetinib show that it has a significant effect in the treatment of cholangiocarcinoma. In the FIGHT-202 clinical trial, pemetinib was administered to 107 patients with previously treated locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements. Patients received 13.5 mg of pemetinib once daily for 14 days, followed by 7 days off treatment, in 21-day cycles until disease progression or unacceptable toxicity. The trial results showed that the overall response rate of pemetinib was 36%, including 2.8% complete response and 33% partial response. This means that most patients experience a reduction in tumor size or improvement in symptoms after treatment. In addition, the disease control rate in this trial was as high as 82%, showing that pemetinib can effectively control the disease in most patients.

InFIGHT-202 trial, pemetinib also showed good safety and tolerability. The most common treatment-emergent adverse reaction (TEAE) is hyperphosphatemia, and other more frequent TEAEs include alopecia, diarrhea, fatigue, dysphagia, nausea, constipation, stomatitis, dry mouth, and decreased appetite. Most of these TEAEs were grade ≤2, and most patients could be relieved by dose adjustment or symptomatic treatment.

In addition toFIGHT-202 trial, pemetinib has also performed well in other clinical trials. For example, in a multi-center, open-label, single-arm trial conducted in China, the objective response rate of pemetinib reached half, demonstrating its efficacy and safety in Chinese patients with similar cholangiocarcinoma.

In conclusion, the clinical trial results of pemetinib show that it, as a targeted drug, has a significant effect in treating patients with cholangiocarcinoma carryingFGFR2 gene fusion or rearrangement. Its high response rate and good safety profile make it a new treatment option for these patients. However, it should be noted that pemetinib is not suitable for all patients with cholangiocarcinoma, and its use should be based on individualized decisions based on the patient's genetic status and physical condition.

Currently, pemetinib, as the original drug, is relatively expensive on the market, with a price tag of tens of thousands of yuan per box, which poses a considerable financial burden to many patients. However, thankfully, Lucius Pharmaceuticals has successfully developed a more affordable generic version of pemetinib, and patients should choose it themselves.