Innovative therapy targeting FGF receptor Incyte cholangiocarcinoma is qualified for priority review

Today, Incyte announced that FDA has accepted its new drug application (NDA) for pemigatinib, a FGFR1 / 2 / 3 inhibitor, for the treatment of locally advanced or metastatic intrahepatic cholangiocarcinoma (ICC) patients carrying FGFR2 gene fusion or rearrangement The FDA also granted the application a priority review qualification and is expected to respond by May 30 next year Cholangiocarcinoma is a rare malignant tumor, which can be divided into ICC and extrahepatic cholangiocarcinoma The existing local treatment options include surgery, radiotherapy, and other ablation therapies The prognosis of patients with advanced cholangiocarcinoma is poor, and there is no approved systemic therapy available Patients also have very limited chemotherapy options Fibroblast growth factor receptor (FGFRs) plays an important role in the proliferation, survival, migration and angiogenesis of tumor cells The fusion, rearrangement, translocation and amplification of FGFR gene are closely related to the occurrence of various cancers The fusion or rearrangement of FGFR2 gene occurs almost exclusively in ICC, accounting for 10% to 16% of patients with ICC Pemigatinib is an oral small molecule inhibitor targeting FGFR1, 2 and 3 Its preclinical data reflect the excellent efficacy and safety for tumors with FGFR gene mutation Previously, the FDA has granted pemigatinib a breakthrough treatment for cholangiocarcinoma and orphan drug qualification Photo source: Incyte official website This NDA submission is based on data on the efficacy and safety of pemigatinib in the treatment of locally advanced or metastatic intrahepatic cholangiocarcinoma patients in a phase 2 trial called light-202 The results showed that in the subgroup of patients with FGFR2 gene fusion or rearrangement, pemigatinib single drug treatment made the patients achieve 36% objective response rate (ORR) and 7.5 months median response time (DOR) "For patients with cholangiocarcinoma, treatment options other than first-line chemotherapy are very limited and often face poor prognosis," said Peter, vice president of Incyte Dr Langmuir said: "FDA accepts the NDA of pemigatinib and grants it the priority review qualification, which we think is very important for patients with locally advanced or metastatic cholangiocarcinoma who have received other therapies and carried FGFR2 gene fusion or rearrangement We look forward to working closely with FDA to bring this innovative targeted therapy to patients with devastating diseases as soon as possible " reference material: [1] Incyte Announces Acceptance and Priority Review of NDA for Pemigatinib as a Treatment for Patients with Cholangiocarcinoma， Retrieved November 27, 2019, from https:// A kind of