European Commission approves BALVERSA®▼ (erdafitinib) for adult patients with unresectable or metastatic urothelial carcinoma

First pan FGFR kinase inhibitor to be approved in the European Economic Area, for adults with unresectable or metastatic urothelial carcinoma and susceptible FGFR3 alterations

Approval based on THOR results, showing 36 percent reduction in risk of death with erdafitinib versus chemotherapy1

BEERSE, BELGIUM (23 August 2024) – Janssen-Cilag International NV, a Johnson & Johnson company, today announced that the European Commission (EC) has approved BALVERSA®▼(erdafitinib) as a once-daily oral monotherapy for the treatment of adult patients with unresectable or metastatic urothelial carcinoma (mUC), harbouring susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations who have previously received at least one line of therapy containing a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor in the unresectable or metastatic treatment setting.2 “Bladder cancer is one of Europe’s most common cancers and the need for innovative treatment options for people living with unresectable or metastatic urothelial carcinoma remains high,” said Yohann Loriot, M.D., Ph.D., Institut Gustave Roussy and University of Paris-Saclay, France.* “Erdafitinib is a novel, targeted therapy that has been shown to significantly improve overall and progression-free survival for patients with FGFR3 alterations, who, until now, have had limited options available.” Europe has the highest rate of bladder cancer compared to all continents globally, with nearly a quarter of a million people diagnosed in 2022,3 representing a 10 percent increase from 2020.4 Urothelial carcinoma (UC) is the most common form of bladder cancer,5 and up to 20 percent of patients with mUC have FGFR alterations.6 Prognosis remains particularly poor for patients with mUC, with only eight percent of people diagnosed at a late metastatic stage surviving for five years.7,8 "This important milestone emphasises the vital role of targeted therapies in addressing the unique genetic and disease characteristics of patients living with urothelial cancer, and reinforces our dedication to advancing cutting-edge, precision treatments in oncology,” said Henar Hevia, Ph.D., Senior Director, EMEA Therapeutic Area Lead, Oncology, Johnson & Johnson Innovative Medicine. “The approval of erdafitinib as a precision therapy further highlights the importance of FGFR testing for all patients with metastatic urothelial cancer, and the need for a multi-disciplinary team approach to optimise outcomes for each patient.” Erdafitinib received EC approval based on results from Cohort 1 of the Phase 3 THOR study (NCT03390504), evaluating the efficacy and safety of erdafitinib (n=136) versus chemotherapy (n=130) in patients with advanced or mUC with select FGFR alterations who have progressed on or after one or two prior treatments, at least one of which includes an anti-PD-(L)1 agent.1,9 In June 2023, based on the recommendation of the independent data safety monitoring committee, the THOR study was stopped following the interim efficacy analysis and all patients randomised to chemotherapy (docetaxel or vinflunine) were offered the opportunity to receive erdafitinib as crossover therapy.1 The results demonstrate that a median overall survival (OS) of over one year was achieved in patients receiving erdafitinib at the data cut-off, marking a significant improvement as compared to those in the chemotherapy arm (12.1 months vs. 7.8 months; hazard ratio [HR]=0.64; 95 percent confidence interval [CI]**, 0.44-0.93; P=0.0050).2 Treatment with erdafitinib also showed an improvement in median progression-free survival (PFS) compared to chemotherapy of 5.6 months versus 2.7 months (HR=0.58; 95 percent CI**, 0.41-0.82; P=0.0002) and confirmed overall response rate (ORR) of 35.3 percent versus 8.5 percent.2

Serious treatment-related adverse events (TRAEs) were observed in 13.3 percent of patients who received erdafitinib and 24.1 percent of patients randomised to chemotherapy.1 Grade 3 or higher adverse events (TRAEs) were observed in 45.9 percent of patients on erdafitinib and 46.4 percent on chemotherapy.1 Amongst patients who received erdafitinib, 8.1 percent had TRAEs that led to discontinuation of therapy, versus 13.4 percent of patients who received chemotherapy.1 TRAEs leading to death were reported in one patient who received erdafitinib and six patients who received chemotherapy.1

“The EC approval of erdafitinib reflects our unwavering commitment to transforming outcomes for people living with unresectable or metastatic urothelial carcinoma,” said Kiran Patel, M.D., Vice President, Clinical Development, Solid Tumours, Johnson & Johnson Innovative Medicine. “We look forward to continuing our research and development efforts to bring new hope and improved outcomes to more patients in the future.”