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Journal Articles New England Journal of Medicine Year : 2019

Erdafitinib in Locally Advanced or Metastatic Urothelial Carcinoma


Alterations in the gene encoding fibroblast growth factor receptor (FGFR) are common in urothelial carcinoma and may be associated with lower sensitivity to immune interventions. Erdafitinib, a tyrosine kinase inhibitor of FGFR1-4, has shown antitumor activity in preclinical models and in a phase 1 study involving patients with FGFR alterations. Methods: In this open-label, phase 2 study, we enrolled patients who had locally advanced and unresectable or metastatic urothelial carcinoma with prespecified FGFR alterations. All the patients had a history of disease progression during or after at least one course of chemotherapy or within 12 months after neoadjuvant or adjuvant chemotherapy. Prior immunotherapy was allowed. We initially randomly assigned the patients to receive erdafitinib in either an intermittent or a continuous regimen in the dose-selection phase of the study. On the basis of an interim analysis, the starting dose was set at 8 mg per day in a continuous regimen (selected-regimen group), with provision for a pharmacodynamically guided dose escalation to 9 mg. The primary end point was the objective response rate. Key secondary end points included progression-free survival, duration of response, and overall survival.

Dates and versions

hal-02930806 , version 1 (04-09-2020)



Yohann Loriot, Andrea Necchi, Se Hoon Park, Jesus Garcia-Donas, Robert Huddart, et al.. Erdafitinib in Locally Advanced or Metastatic Urothelial Carcinoma. New England Journal of Medicine, 2019, 381 (4), pp.338-348. ⟨10.1056/NEJMoa1817323⟩. ⟨hal-02930806⟩
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