{"entity":{"id":"erdafitinib","kind":"drug","name":"Erdafitinib","aka":[],"tldr":"The first targeted pill for bladder cancer, for the roughly 20% of tumours with FGFR3 alterations, used after immunotherapy.","summary":"Erdafitinib is an oral pan-FGFR (1 to 4) tyrosine kinase inhibitor for the roughly 20% of urothelial cancers with FGFR3 or FGFR2 alterations, so molecular testing is required before use. It received accelerated approval in 2019 on BLC2001 (ORR 40%) and full approval in January 2024 on THOR cohort 1, where it gave OS 12.1 versus 7.8 months (HR 0.64) against chemotherapy after platinum and immunotherapy. THOR cohort 2, against pembrolizumab in immunotherapy-naive patients, showed no benefit, so the drug is positioned after a PD-1 or PD-L1 inhibitor. Hyperphosphataemia (79%), stomatitis (58%) and central serous retinopathy (17%) need monitoring, including regular eye examinations. Johnson & Johnson develops it. It is the first targeted pill in bladder cancer, and it only works when the tumour carries the matching alteration.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Erdafitinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Erdafitinib"}],"tags":[],"related":["fgfr3-alteration"],"cancers":["urothelial","muscle-invasive-bladder-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["fgfr2"],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":["fgfr-signalling"],"terms":["fgfr3","ocular-toxicity"],"trials":["thor","nct05316155","nct02365597","nct03473743"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Balversa","modality":"Small-molecule kinase inhibitor (pan-FGFR)","mechanism":"Pan-FGFR (1-4) tyrosine kinase inhibitor.","approvals":[{"region":"US","year":2019,"indication":"FGFR3/2-altered advanced urothelial cancer after platinum (accelerated)"},{"region":"US","year":2024,"indication":"FGFR3-altered advanced urothelial cancer after ≥1 systemic therapy incl. PD-1/PD-L1 (full)"}],"mechanismSteps":["Oral absorption; dose titrated to serum phosphate","Blocks FGFR3 (mutated or fused) autophosphorylation","Shuts down MAPK and PI3K signalling in FGFR-driven luminal-papillary tumours","Resistance via gatekeeper mutations and pathway bypass"],"dosing":{"route":"Oral","schedule":"8 mg daily, up-titrated to 9 mg if serum phosphate <5.5 mg/dL at day 14","modifications":"Hold for phosphate >7 mg/dL or ocular toxicity","monitoring":"Phosphate monthly; ophthalmology exam monthly for 4 months"},"toxicity":[{"event":"Hyperphosphataemia","anyGradePct":79},{"event":"Stomatitis","anyGradePct":58},{"event":"Diarrhoea","anyGradePct":55},{"event":"Central serous retinopathy","anyGradePct":17},{"event":"Onycholysis","anyGradePct":23}],"access":[],"regulatoryEvents":[{"date":"2019-04-12","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 urothelial carcinoma (mUC), that has: susceptible FGFR3 or FGFR2 genetic alterations, and progressed during or following at least one line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy. Select patients for therapy based on an FDA-approved companion diagnostic for BALVERSA."},{"date":"2024-01-19","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2019 converted to traditional approval 4.8 years after it was granted.","indication":"Treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC), that has: susceptible FGFR3 or FGFR2 genetic alterations, and progressed during or following at least one line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy. Select patients for therapy based on an FDA-approved companion diagnostic for BALVERSA."}]},"route":"/drugs/erdafitinib/","neighbours":{"biomarker":[{"id":"fgfr3-alteration","kind":"biomarker","name":"FGFR3 alteration (mutation or fusion)","route":"/biomarkers/fgfr3-alteration/"}],"cancer":[{"id":"urothelial","kind":"cancer","name":"Bladder & urothelial cancer","route":"/cancers/urothelial/"},{"id":"muscle-invasive-bladder-cancer","kind":"cancer","name":"Muscle-invasive and advanced bladder cancer","route":"/cancers/muscle-invasive-bladder-cancer/"}],"technology":[{"id":"kinase-inhibitors","kind":"technology","name":"Small-molecule kinase inhibitors","route":"/technologies/kinase-inhibitors/"}],"target":[{"id":"fgfr1","kind":"target","name":"FGFR1","route":"/targets/fgfr1/"},{"id":"fgfr2","kind":"target","name":"FGFR2","route":"/targets/fgfr2/"},{"id":"fgfr3-receptor","kind":"target","name":"FGFR3","route":"/targets/fgfr3-receptor/"}],"company":[{"id":"johnson-johnson","kind":"company","name":"Johnson & Johnson","route":"/companies/johnson-johnson/"}],"pathway":[{"id":"bladder-cancer-signalling","kind":"pathway","name":"Bladder cancer (KEGG map)","route":"/pathways/bladder-cancer-signalling/"},{"id":"fgfr-signalling","kind":"pathway","name":"FGF / FGFR signalling","route":"/pathways/fgfr-signalling/"},{"id":"proteoglycans-in-cancer","kind":"pathway","name":"Proteoglycans in cancer","route":"/pathways/proteoglycans-in-cancer/"}],"term":[{"id":"fgfr3","kind":"term","name":"FGFR3 alterations (bladder cancer)","route":"/terms/fgfr3/"},{"id":"ocular-toxicity","kind":"term","name":"Ocular toxicity (keratopathy, blurred vision)","route":"/terms/ocular-toxicity/"}],"trial":[{"id":"nct03473743","kind":"trial","name":"A Study of Erdafitinib in Participants With Metastatic or Locally Advanced Urothelial Cancer","route":"/trials/nct03473743/"},{"id":"nct02365597","kind":"trial","name":"An Efficacy and Safety Study of Erdafitinib (JNJ-42756493) in Participants With Urothelial Cancer","route":"/trials/nct02365597/"},{"id":"pediatric-match","kind":"trial","name":"NCI-COG Pediatric MATCH (APEC1621)","route":"/trials/pediatric-match/"},{"id":"nct05316155","kind":"trial","name":"Study of Erdafitinib Intravesical Delivery System for Localized Bladder Cancer","route":"/trials/nct05316155/"},{"id":"thor","kind":"trial","name":"THOR","route":"/trials/thor/"}],"drug":[{"id":"therascreen-cdx","kind":"drug","name":"therascreen companion diagnostic kits (KRAS, EGFR, PIK3CA, FGFR, BRAF)","route":"/drugs/therascreen-cdx/"}]}}