Press ReleasJanssen Submits Marketing Authorisation Application to the European Medicines Agency Seeking Approval of Erdafitinib for the Treatment of Patients with Locally Advanced or Metastatic Urothelial Cancer with Susceptible FGFR Alterations

Pending approval, erdafitinib, an investigational, once-daily oral pan-fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor will become the first therapy targeting FGFR alterations in patients with metastatic urothelial carcinoma, one of Europe’s most common cancers

The submission is based on results from the Phase 3 THOR study, which were featured in a Late-Breaking Presentation Session at the 2023 ASCO Annual Meeting in June

8 September 2023 -- Beerse, Belgium -- The Janssen Pharmaceutical Companies of Johnson & Johnson announced today the submission of 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 world, with more than 203,000 patients diagnosed in 2020 alone. UC is the most common form and up to 20 percent of patients with metastatic UC (mUC) have FGFR alterations. Patients with mUC, including FGFR-driven tumours, face a particularly poor prognosis and the need for innovative therapies remains high. Only eight percent of people diagnosed at a late, metastatic stage will survive for five years or more.

“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.”

This MAA is supported by data from Cohort 1 of the randomised, controlled, open-label, multicentre Phase 3 THOR study (NCT03390504), evaluating the efficacy and safety of erdafitinib versus chemotherapy. 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. 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.The safety profile of erdafitinib observed in THOR was consistent with the previously reported safety profile of erdafitinib in metastatic urothelial carcinoma (mUC). These pivotal data from THOR were featured in a Late-Breaking Presentation Session (Abstract #LBA4619) at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting.

“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, MD, vice president, Clinical Development, Solid Tumors, 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.”

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. 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 3 THOR study.

Visit: www.janssen.com/

About Janssen
At Janssen, we're creating a future where disease is a thing of the past. We're the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular, Metabolism & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.
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