Full enrollment results from cohort 1 of the phase III randomized THOR trial of erdafitinib versus chemotherapy in FGFR-altered advanced urothelial carcinoma
In brief
Erdafitinib extended median survival by 3.4 months in advanced urothelial cancer
In the randomized THOR trial, patients with advanced urothelial cancer and susceptible FGFR alterations lived a median 12.1 months with erdafitinib, versus 8.7 months with chemotherapy, after prior immunotherapy. Tumors shrank in 47% versus 13%, while severe side effects were similarly common; longer follow-up confirmed the survival benefit, though treatment choice still requires weighing risks for each patient.
- Journal
- ESMO open (Q1)
- Published
- 30 September 2026
- Study design
- Randomized controlled trial
- Evidence level
- Level 1, High (CEBM 1b)
- Authors
- Y Loriot, N Matsubara, S H Park, R A Huddart, E F Burgess, N Houede, et al.
- PMID
- 42815410
- DOI
- 10.1016/j.esmoop.2026.108583
Why clinicians should know about it
- Picked for Oncology and Radiation Oncology (paper of the day, 1 October 2026): Phase III RCT, OS benefit of erdafitinib versus chemotherapy
Abstract
BACKGROUND: Cohort 1 of the phase III THOR trial met the primary endpoint of significantly longer overall survival (OS) with erdafitinib versus chemotherapy in patients with metastatic urothelial carcinoma (mUC) and select FGFR3/2 alterations who have progressed after prior anti-PD-(L)1 treatment. We present efficacy and safety results for the full cohort 1 at the end-of-study data cut-off (final database lock: 7 June 2024). PATIENTS AND METHODS: Adults with mUC with susceptible FGFR3/2 alterations who had progression after 1-2 prior treatments that included an anti-PD-(L)1 were randomly assigned 1 : 1 to receive erdafitinib or investigator's choice of chemotherapy (docetaxel or vinflunine). The primary endpoint was OS and progression-free survival (PFS) and objective response rate (ORR) were key secondary endpoints. RESULTS: At the end of data collection, median follow-up was 21.0 months for erdafitinib and 18.0 months for chemotherapy for the 278 patients in the fully enrolled THOR cohort 1 (erdafitinib, n = 143; chemotherapy, n = 135). Median OS remained significantly longer with erdafitinib versus chemotherapy [12.1 versus 8.7 months, hazard ratio (HR) 0.65, 95% confidence interval (CI) 0.48-0.86, P = 0.0031]. Erdafitinib also showed sustained improvements over chemotherapy in PFS (median 5.4 versus 2.7 months, HR 0.59, 95% CI 0.45-0.77, P < 0.0001) and ORR (46.9% versus 12.6%, relative risk 3.74, 95% CI 2.31-6.04, P < 0.001). Grade 3-4 treatment-emergent adverse events occurred in 67.6% of patients in the erdafitinib group and 65.8% in the chemotherapy group. Erdafitinib was associated with fewer treatment-related serious adverse events (AEs) (12.7% versus 24.8%), treatment-related deaths (0.7% versus 6.0%), and treatment-related AEs that led to discontinuations (9.2% versus 14.5%) compared with chemotherapy. CONCLUSIONS: At the end of data collection, erdafitinib continued to demonstrate survival and clinical response benefit versus chemotherapy, consistent with the primary analysis. No new safety signals were observed. These mature data support the use of erdafitinib in patients with advanced/mUC and susceptible FGFR3 alterations after PD-(L)1 inhibition.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.