Balversa by J&J approved in EU as first FGFR3-targeted bladder cancer therapy

The European Commission has approved Johnson & Johnson's Balversa (erdafitinib) for use in adults with unresectable or metastatic urothelial carcinoma (mUC) that exhibit susceptible FGFR3 genetic alterations. This approval marks the first time a pan-FGFR kinase inhibitor has been cleared for this patient group in the EU. The decision follows Balversa's full FDA approval in January for a similar condition.

The EU's endorsement comes after a positive recommendation from the European Medicines Agency's (EMA) drug advisory committee in June. The approval is supported by data from Cohort 1 of the Phase III THOR study, which focused on patients whose disease had progressed after one or two previous treatments, at least one of which was an anti-PD-(L)1 therapy. Results from this study indicated that Balversa was associated with a 36% reduction in the risk of death compared to chemotherapy. Patients on Balversa achieved a median overall survival of 12.1 months, which is 4.3 months longer than those on chemotherapy. The study also demonstrated improved progression-free survival (5.6 months compared to 2.7 months) and a higher overall response rate (35.3% versus 8.5%) for Balversa.

Henar Hevia, EMEA therapeutic area lead for oncology at Johnson & Johnson Innovative Medicine, emphasized the significance of this milestone, highlighting the critical role of targeted therapies in addressing the specific genetic and disease characteristics of urothelial cancer patients. Hevia also underscored the importance of FGFR testing for all patients with metastatic urothelial cancer.

Bladder cancer rates are highest in Europe compared to other continents, with nearly a quarter of a million new diagnoses in 2022. Johnson & Johnson estimates that up to 20% of patients with mUC have FGFR alterations.

Jose Pablo Maroto Rey, head of the urologic oncology unit at Hospital de la Santa Creu i Sant Pau in Barcelona, identified Astellas and Pfizer's antibody-drug conjugate Padcev (enfortumab vedotin) as the main competitor to Balversa. Maroto Rey pointed out that more research is needed to determine whether patients with FGFR mutations respond differently to other therapies. He suggested that if these patients are poor responders to enfortumab vedotin, they might need to be treated first with FGFR inhibitors. He also noted that future competition would likely come from other drugs developed for third-line treatment.