{"entity":{"id":"mobocertinib","kind":"drug","name":"Mobocertinib","aka":[],"tldr":"Mobocertinib was the first oral drug for EGFR exon 20 insertion lung cancer, approved in 2021 and withdrawn in 2023-24 after its confirmatory trial failed.","summary":"Mobocertinib is a covalent EGFR inhibitor designed for exon 20 insertion mutations, which resist earlier EGFR TKIs, while sparing wild-type EGFR relative to those agents. It received accelerated US approval in 2021 on a 28% response rate in patients who had progressed after platinum chemotherapy, the first oral drug for this subgroup. The confirmatory EXCLAIM-2 trial (2023) failed to beat platinum chemotherapy in the first-line setting, and Takeda withdrew the drug globally, with the US withdrawal completed in 2024. Diarrhoea and QT prolongation were prominent toxicities. Amivantamab and sunvozertinib now cover the exon 20 insertion indication. Mobocertinib shows how an accelerated approval can be reversed when the confirmatory trial does not deliver.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Mobocertinib","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Mobocertinib"}],"tags":["gap-fill"],"related":[],"cancers":["nsclc","egfr-mutant-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr"],"drugs":[],"companies":["takeda"],"institutions":[],"pathways":[],"terms":["egfr-exon20-insertion"],"trials":["nct04129502"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Exkivity","modality":"Small-molecule EGFR exon 20 insertion TKI (irreversible)","mechanism":"Covalent EGFR inhibitor designed for exon 20 insertion mutations, sparing wild-type EGFR relative to earlier TKIs.","approvals":[{"region":"US","year":2021,"indication":"EGFR exon 20 insertion NSCLC after platinum","note":"Withdrawn 2023-24"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2021-09-15","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy"},{"date":"2024-07-15","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 2.8 years after its accelerated approval.","indication":"Treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy"}]},"route":"/drugs/mobocertinib/","neighbours":{"cancer":[{"id":"egfr-mutant-nsclc","kind":"cancer","name":"EGFR-mutated non-small-cell lung cancer","route":"/cancers/egfr-mutant-nsclc/"},{"id":"lung-cancer","kind":"cancer","name":"Lung cancer (all types)","route":"/cancers/lung-cancer/"},{"id":"nsclc","kind":"cancer","name":"Non-small-cell lung cancer","route":"/cancers/nsclc/"}],"technology":[{"id":"kinase-inhibitors","kind":"technology","name":"Small-molecule kinase inhibitors","route":"/technologies/kinase-inhibitors/"}],"target":[{"id":"egfr","kind":"target","name":"EGFR","route":"/targets/egfr/"}],"company":[{"id":"takeda","kind":"company","name":"Takeda","route":"/companies/takeda/"}],"term":[{"id":"egfr-exon20-insertion","kind":"term","name":"EGFR exon 20 insertion","route":"/terms/egfr-exon20-insertion/"},{"id":"lung-failed-programmes","kind":"term","name":"Lung cancer: the failed and stopped programmes, and why","route":"/terms/lung-failed-programmes/"}],"trial":[{"id":"nct04129502","kind":"trial","name":"TAK-788 as First-Line Treatment Versus Platinum-Based Chemotherapy for Non-Small Cell Lung Cancer (NSCLC) With EGFR Exon 20 Insertion Mutations","route":"/trials/nct04129502/"}],"biomarker":[{"id":"egfr-exon-20-insertion","kind":"biomarker","name":"EGFR exon 20 insertion","route":"/biomarkers/egfr-exon-20-insertion/"}]}}