{"entity":{"id":"infigratinib","kind":"drug","name":"Infigratinib","aka":["BGJ398"],"tldr":"Infigratinib is an FGFR inhibitor pill given accelerated approval in the United States in 2021 for bile duct cancer with an FGFR2 fusion, then withdrawn in 2024 when its confirmatory trial could not enrol; it is now being developed for achondroplasia instead.","summary":"Infigratinib, discovered at Novartis and developed by BridgeBio's QED Therapeutics with Helsinn, received FDA accelerated approval in May 2021 for previously treated, unresectable or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement, on a response rate of about 23 percent in a single-arm phase 2 trial. The first-line confirmatory trial PROOF 301 struggled to recruit against pemigatinib and futibatinib, and the sponsors withdrew the cholangiocarcinoma indication in 2024. Hyperphosphataemia, eye and nail changes are the FGFR class effects. BridgeBio continues the molecule at low dose for children with achondroplasia.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Infigratinib","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Infigratinib"},{"label":"ChEMBL CHEMBL1852688","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1852688"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["cholangiocarcinoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["fgfr2"],"drugs":["pemigatinib","futibatinib"],"companies":["bridgebio-oncology-therapeutics","helsinn"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02159066","nct01975701","nct02706691"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Truseltiq","modality":"Oral FGFR1 to 3 kinase inhibitor","mechanism":"An ATP-competitive inhibitor of FGFR1, FGFR2 and FGFR3 kinases; tumours driven by FGFR2 fusions depend on that signalling and shrink when it is blocked.","approvals":[{"region":"US","year":2021,"indication":"Previously treated unresectable or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement (accelerated approval)","note":"Indication withdrawn in 2024"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2021-05-28","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/withdrawn-fda-grants-accelerated-approval-infigratinib-metastatic-cholangiocarcinoma","indication":"Treatment of adult patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangement as detected by an FDA approved test"},{"date":"2024-05-16","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 3.0 years after its accelerated approval.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/withdrawn-fda-grants-accelerated-approval-infigratinib-metastatic-cholangiocarcinoma","indication":"Treatment of adult patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangement as detected by an FDA approved test"}]},"route":"/drugs/infigratinib/","neighbours":{"cancer":[{"id":"cholangiocarcinoma","kind":"cancer","name":"Biliary tract cancer (cholangiocarcinoma)","route":"/cancers/cholangiocarcinoma/"}],"technology":[{"id":"kinase-inhibitors","kind":"technology","name":"Small-molecule kinase inhibitors","route":"/technologies/kinase-inhibitors/"}],"target":[{"id":"fgfr2","kind":"target","name":"FGFR2","route":"/targets/fgfr2/"}],"drug":[{"id":"futibatinib","kind":"drug","name":"Futibatinib","route":"/drugs/futibatinib/"},{"id":"pemigatinib","kind":"drug","name":"Pemigatinib","route":"/drugs/pemigatinib/"}],"company":[{"id":"bridgebio-oncology-therapeutics","kind":"company","name":"BridgeBio Oncology Therapeutics","route":"/companies/bridgebio-oncology-therapeutics/"},{"id":"helsinn","kind":"company","name":"Helsinn","route":"/companies/helsinn/"}],"trial":[{"id":"nct01975701","kind":"trial","name":"A Phase 2 Study of BGJ398 in Patients With Recurrent GBM","route":"/trials/nct01975701/"},{"id":"nct02706691","kind":"trial","name":"BGJ398 in Treating Patients With FGFR Positive Recurrent Head and Neck Cancer","route":"/trials/nct02706691/"},{"id":"nct02159066","kind":"trial","name":"LGX818 and MEK162 in Combination With a Third Agent (BKM120, LEE011, BGJ398 or INC280) in Advanced BRAF Melanoma","route":"/trials/nct02159066/"}],"biomarker":[{"id":"fgfr2-fusion-rearrangement","kind":"biomarker","name":"FGFR2 fusion or rearrangement","route":"/biomarkers/fgfr2-fusion-rearrangement/"}]}}