Janssen submits EMA filing for bladder cancer treatment 

Bladder cancer

The Janssen Pharmaceutical Companies of Johnson & Johnson has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking approval of erdafitinib for the treatment of adult patients with locally advanced unresectable or metastatic urothelial carcinoma (UC), harbouring susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations, with disease progression during or following at least one line of therapy containing a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-[L]1) inhibitor. 

Europe has one of the highest rates of bladder cancer in the world1, with more than 203,000 patients diagnosed in 2020 alone2. UC is the most common form3, and up to 20% of patients with metastatic UC (mUC) have FGFR alterations4. Patients with mUC, including FGFR-driven tumours, face a particularly poor prognosis and the need for innovative therapies remains high.5 Only 8% of people diagnosed at a late, metastatic stage will survive for five years or more.6,7

“For patients with advanced UC, including FGFR-driven tumours, outcomes remain poor and treatment options are limited; therefore, there is a need for novel, targeted therapies,” said Martin Vogel, EMEA Therapeutic Area Lead Oncology, Janssen-Cilag GmbH. “We are excited by the prospect of bringing innovative, personalised approaches to market for patients as we work towards our wider goal of making this complex disease a more manageable and ultimately curable condition.” 

The study  

This MAA is supported by data from Cohort 1 of the randomised, controlled, open-label, multicentre Phase III THOR study (NCT03390504), evaluating the efficacy and safety of erdafitinib versus chemotherapy.7  

The study met its primary endpoint of overall survival (OS), with patients who received erdafitinib achieving a median OS of over one year at the prespecified interim analysis data cutoff.4 As the interim results met the predefined criteria for superiority of treatment with erdafitinib over chemotherapy, the independent data safety monitoring committee recommended that the study be stopped and that patients randomised to chemotherapy be offered the opportunity to cross-over to erdafitinib.4

The safety profile of erdafitinib observed in THOR was consistent with the previously reported safety profile of erdafitinib in metastatic urothelial carcinoma (mUC).2,4 These pivotal data from THOR were featured in a late-breaking presentation session at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting2. 

“This submission, and Janssen’s ongoing study of erdafitinib, reinforces our commitment to deliver much-needed targeted therapies in the areas of high unmet need, including for devastating diseases like metastatic UC,” said Kiran Patel, Vice President, Clinical Development, Solid Tumours, Janssen Research & Development, LLC. “Erdafitinib has demonstrated promising results in advanced, FGFR-altered UC, making this submission a vital step towards improving outcomes for patients in the future. The OS benefit we’ve seen with erdafitinib also supports the need for biomarker testing for FGFR alterations in all patients with metastatic UC.” 

FDA approval  

In April 2019, erdafitinib received accelerated approval from the US Food and Drug Administration (FDA) as a targeted therapy for adult patients with locally advanced or mUC with susceptible FGFR3 or FGFR2 genetic alterations and who have progressed during or following at least one line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.8 On August 29, Janssen submitted a supplemental New Drug Application (sNDA) to the FDA seeking full approval of erdafitinib in this indication based on Cohort 1 of the Phase III THOR study. 


  1. Wong MCS, et al. The global epidemiology of bladder cancer: a joinpoint regression analysis of its incidence and mortality trends and projection. Scientific Reports. 2018;8:1129. 
  2. Loriot Y, et al. Phase 3 THOR study: results of erdafitinib versus chemotherapy in patients with advanced or metastatic urothelial cancer with select fibroblast growth factor receptor alterations. Oral presentation at ASCO 2023 
  3. www.cancer.net/cancer-types/bladder-cancer/introduction#:~:text=Urothelial%20carcinoma%20(or%20UCC)%20accounts,that%20line%20 the%20urinary%20tract  
  4. Loriot Y, et al. Erdafitinib in Locally Advanced or Metastatic Urothelial Carcinoma. N Engl J Med 2019; 381(4):338-348. 
  5. Fernandez E, et al. Prognostic value and clinical significance of FGFR genomic alterations (GAs) in metastatic urothelial cancer patients. J Clin Med. 2022;11(15): 4483. 
  6. Montazeri K & Bellmunt J. Erdafitinib for the treatment of metastatic bladder cancer. Expert Review of Clinical Pharmacology. 2020;13(1):1-6. 
  7. https://classic.clinicaltrials.gov/ct2/show/NCT03390504 
  8. https://www.fda.gov/news-events/press-announcements/fda-approves-first-targeted-therapy-metastatic-bladder-cancer  

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