Erdafitinib Approved in the UK for Bladder Cancer with FGFR3 Alterations

• The MHRA has approved erdafitinib for adults with unresectable or metastatic urothelial carcinoma (UC) harboring FGFR3 genetic alterations, offering a new treatment option.
• Erdafitinib, an oral FGFR kinase inhibitor, demonstrated extended overall survival compared to chemotherapy in the Phase 3 THOR study, with a median overall survival of 12.1 months versus 7.8 months.
• The approval provides hope for approximately 20% of bladder cancer patients in the UK who have FGFR3 genetic alterations driving cancer growth, addressing a critical unmet need.
• Johnson & Johnson is committed to working with health authorities to make erdafitinib available through the NHS, ensuring access to this innovative precision therapy for eligible patients.

The UK's Medicines and Healthcare products Regulatory Agency (MHRA) has approved erdafitinib (BALVERSA, Johnson & Johnson) for the treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC) who harbor FGFR3 genetic alterations and have previously undergone at least one line of therapy containing a PD-1 or PD-L1 inhibitor. This approval marks a significant advancement in the treatment landscape for advanced bladder cancer.

This decision provides a new therapeutic avenue for a subset of bladder cancer patients with specific genetic profiles. In the UK, approximately 10,500 individuals are diagnosed with bladder cancer each year, with about 20% exhibiting FGFR3 genetic alterations that promote cancer development.

Clinical Efficacy

The MHRA's approval was primarily based on the results of the Phase 3 THOR study. The study demonstrated a statistically significant improvement in overall survival (OS) for patients treated with erdafitinib compared to those receiving chemotherapy. Specifically, the median overall survival was 12.1 months in the erdafitinib arm versus 7.8 months in the chemotherapy arm.

Progression-free survival (PFS) also favored erdafitinib, with a median PFS of 5.6 months compared to 2.7 months for chemotherapy.

Mechanism of Action

Erdafitinib is an oral FGFR kinase inhibitor that targets specific genetic alterations in the FGFR3 gene, which are present in a subset of urothelial carcinomas. By inhibiting FGFR3, erdafitinib disrupts cancer cell growth and proliferation, leading to improved clinical outcomes.

Expert Commentary

Professor Alison Birtle of Lancashire Teaching Hospitals NHS Foundation Trust highlighted the importance of this approval, stating, “Patients living with this advanced stage of bladder cancer, and whose tumours harbour FGFR3 alterations, require access to innovative precision therapies that can target the specific characteristics of their disease. Today’s authorisation of erdafitinib will come as welcome news to eligible patients.”

Future Availability

Johnson & Johnson aims to collaborate with health authorities to ensure erdafitinib is accessible through the NHS as quickly as possible, offering new hope to patients with advanced bladder cancer.