{"entity":{"id":"pralsetinib","kind":"drug","name":"Pralsetinib","aka":[],"tldr":"Pralsetinib is the second selective RET pill for lung cancer, less used than selpercatinib after a change of owner and label.","summary":"Pralsetinib is a selective RET inhibitor that blocks the RET kinase in tumours driven by RET fusions or mutations while sparing most other kinases. It is used for RET-fusion metastatic non-small-cell lung cancer, where ARROW reported a response rate of about 72% in treatment-naive patients. It was approved in 2020, with full approval in 2023; the thyroid indications were withdrawn in the US in 2023 after Roche returned rights to Blueprint (now Rigel). It remains an option in RET-fusion NSCLC but is less used than selpercatinib, which has randomised phase 3 support and a broader label. For a newcomer, pralsetinib is the second of two RET-selective pills, effective but overshadowed by commercial and label changes rather than by weak efficacy.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Pralsetinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Pralsetinib"},{"label":"NICE TA812: pralsetinib for treating RET fusion-positive advanced non-small-cell lung cancer (not recommended)","url":"https://www.nice.org.uk/guidance/ta812"}],"tags":[],"related":["ret-fusion"],"cancers":["nsclc","medullary-thyroid-cancer","ret-fusion-nsclc","lung-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["ret"],"drugs":["selpercatinib"],"companies":["blueprint-medicines","rigel-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04302025","arrow-thyroid"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Gavreto","modality":"Small-molecule kinase inhibitor (RET)","mechanism":"Selective RET inhibitor.","approvals":[{"region":"US","year":2020,"indication":"RET-fusion metastatic NSCLC (full approval 2023)"},{"region":"England (NICE)","year":2022,"indication":"RET fusion-positive advanced non-small-cell lung cancer in patients who have not had a RET inhibitor: not recommended","note":"TA812, published 3 August 2022, does not recommend pralsetinib within its marketing authorisation, so it is not routinely funded in England."}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2020-09-04","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test"},{"date":"2020-12-01","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy"},{"date":"2020-12-01","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 5.8 years later, when the FDA's table was read.","indication":"Treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate)"},{"date":"2023-07-20","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 2.6 years after its accelerated approval.","indication":"Treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy"},{"date":"2023-08-09","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2020 converted to traditional approval 2.9 years after it was granted.","indication":"Treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test"}]},"route":"/drugs/pralsetinib/","neighbours":{"biomarker":[{"id":"ret-fusion","kind":"biomarker","name":"RET fusion and RET mutation","route":"/biomarkers/ret-fusion/"}],"cancer":[{"id":"lung-cancer","kind":"cancer","name":"Lung cancer (all types)","route":"/cancers/lung-cancer/"},{"id":"medullary-thyroid-cancer","kind":"cancer","name":"Medullary thyroid cancer","route":"/cancers/medullary-thyroid-cancer/"},{"id":"multiple-endocrine-neoplasia","kind":"cancer","name":"Multiple endocrine neoplasia syndromes (MEN1, MEN2, MEN4)","route":"/cancers/multiple-endocrine-neoplasia/"},{"id":"nsclc","kind":"cancer","name":"Non-small-cell lung cancer","route":"/cancers/nsclc/"},{"id":"ret-fusion-nsclc","kind":"cancer","name":"RET fusion-positive non-small-cell lung cancer","route":"/cancers/ret-fusion-nsclc/"},{"id":"thyroid","kind":"cancer","name":"Thyroid cancer","route":"/cancers/thyroid/"}],"technology":[{"id":"kinase-inhibitors","kind":"technology","name":"Small-molecule kinase inhibitors","route":"/technologies/kinase-inhibitors/"}],"target":[{"id":"ret","kind":"target","name":"RET","route":"/targets/ret/"}],"drug":[{"id":"oncomine-dx-target-test","kind":"drug","name":"Oncomine Dx Target Test","route":"/drugs/oncomine-dx-target-test/"},{"id":"selpercatinib","kind":"drug","name":"Selpercatinib","route":"/drugs/selpercatinib/"}],"company":[{"id":"blueprint-medicines","kind":"company","name":"Blueprint Medicines","route":"/companies/blueprint-medicines/"},{"id":"cstone","kind":"company","name":"CStone Pharmaceuticals","route":"/companies/cstone/"},{"id":"rigel-pharmaceuticals","kind":"company","name":"Rigel Pharmaceuticals","route":"/companies/rigel-pharmaceuticals/"}],"trial":[{"id":"nct04302025","kind":"trial","name":"A Study of Multiple Therapies in Biomarker-selected Participants With Resectable Stages IB-III Non-small Cell Lung Cancer (NSCLC)","route":"/trials/nct04302025/"},{"id":"arrow-thyroid","kind":"trial","name":"ARROW (thyroid cohorts)","route":"/trials/arrow-thyroid/"}],"term":[{"id":"basket-trial","kind":"term","name":"Basket trial","route":"/terms/basket-trial/"},{"id":"lung-uk-drug-access","kind":"term","name":"Lung cancer drugs in England: what NICE has recommended","route":"/terms/lung-uk-drug-access/"},{"id":"lung-failed-programmes","kind":"term","name":"Lung cancer: the failed and stopped programmes, and why","route":"/terms/lung-failed-programmes/"}],"person":[{"id":"justin-gainor","kind":"person","name":"Justin F. Gainor","route":"/people/justin-gainor/"}],"paper":[{"id":"paper-arrow-pralsetinib-gainor-lancet-oncol-2021","kind":"paper","name":"ARROW: pralsetinib for RET fusion-positive non-small-cell lung cancer","route":"/key-papers/paper-arrow-pralsetinib-gainor-lancet-oncol-2021/"}],"pathway":[{"id":"nsclc-signalling","kind":"pathway","name":"Non-small cell lung cancer (KEGG map)","route":"/pathways/nsclc-signalling/"},{"id":"thyroid-cancer-signalling","kind":"pathway","name":"Thyroid cancer (KEGG map)","route":"/pathways/thyroid-cancer-signalling/"}]}}