MAA is submitted 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 – Janssen Pharmaceutical

The Janssen Pharmaceutical Companies of Johnson & Johnson announced 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.

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. 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.