{"entity":{"id":"paper-libretto-001-selpercatinib-nsclc-nejm-2020","kind":"paper","name":"LIBRETTO-001: selpercatinib in RET fusion-positive non-small-cell lung cancer","aka":[],"tldr":"The selective RET inhibitor selpercatinib shrank tumours in 64 percent of previously treated and 85 percent of untreated patients with RET fusion-positive lung cancer, including brain metastases, and led to the first approval for this driver.","summary":"Phase 1/2 study of 105 previously treated and 39 treatment-naive patients with RET fusion-positive non-small-cell lung cancer treated with selpercatinib.\n\nObjective response was 64 percent in previously treated patients with median duration of response 17.5 months and median progression-free survival 16.5 months; 85 percent in treatment-naive patients; intracranial response 91 percent among evaluable patients. Hypertension, liver enzyme rise and QT prolongation were the main toxicities.","asOf":"2026-09-17","links":[{"label":"N Engl J Med 2020","url":"https://doi.org/10.1056/NEJMoa2005653"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/32846060/"}],"tags":[],"related":[],"cancers":["ret-fusion-nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":["selpercatinib"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["nejm"],"dependsOn":[],"notes":[],"journal":"New England Journal of Medicine","year":2020,"doi":"10.1056/NEJMoa2005653","pmid":"32846060","authors":"Drilon A, Oxnard GR, Tan DSW, et al.","paperType":"observational","findings":["Objective response 64 percent (previously treated) and 85 percent (treatment-naive).","Median progression-free survival 16.5 months in previously treated patients."],"whatItMeans":"RET fusion testing is standard in lung adenocarcinoma and selpercatinib the preferred first-line RET inhibitor, confirmed against chemo-immunotherapy in LIBRETTO-431.","caveats":["Single-arm study.","Hypersensitivity reactions in patients recently treated with immunotherapy."],"changedPractice":true,"participants":144},"route":"/key-papers/paper-libretto-001-selpercatinib-nsclc-nejm-2020/","neighbours":{"cancer":[{"id":"ret-fusion-nsclc","kind":"cancer","name":"RET fusion-positive non-small-cell lung cancer","route":"/cancers/ret-fusion-nsclc/"}],"drug":[{"id":"selpercatinib","kind":"drug","name":"Selpercatinib","route":"/drugs/selpercatinib/"}],"journal":[{"id":"nejm","kind":"journal","name":"New England Journal of Medicine","route":"/journals/nejm/"}]}}