Approvals

BALVERSA (erdafitinib) has been granted marketing approval by the UK Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of the most common type of bladder cancer, metastatic or unresectable urothelial carcinoma. The treatment, produced by Johnson & Johnson, will be a monotherapy for adults with bladder cancer.

The decision by the MHRA was made based on the results from phase 3 of the THOR study. The aim of the study was to evaluate the efficacy and safety of erdafitainib versus chemotherapy in patients with advanced metastatic or unresectable urothelial carcinoma, inclusive of patients with fibroblast growth factor receptor 3 (FGFR3) alterations who have progressed on or after one or two prior treatments, at least one of which includes a PD-1 or PD-L1 inhibitor. THOR was a multicentre, open-label randomised trial with a primary endpoint of overall survival (OS). The second primary endpoint was objective response rate, progression-free survival (PFS) and duration of response. In June 2023, the study was stopped based on the recommendation of an independent data safety monitoring committee at the interim analysis in order to allow patients the opportunity to cross over from chemotherapy to erdafitinab, despite adverse events of any cause occurring in 98.5% of the patients in the erdafitinib group and 97.3% of those in the chemotherapy group.

Dr John Fleming, country medical director, Johnson &Johnson Innovative Medicine UK stated, “We are delighted that the MHRA has recognised the value that erdafitinib could bring to eligible patients with metastatic urothelial cancer. This milestone reflects J&J’s long-standing dedication to getting in front of cancer and delivering the most innovative precision therapies to patients in need. We look forward to progressing with HTA submissions for erdafitinib in the coming months, with the view to enabling eligible patients to access erdafitinib through the NHS as soon as possible.”

BALVERSA is a once-daily oral FGFR kinase inhibitor and has been shown to extend OS in patients compared to chemotherapy in the second-line setting.

Professor Alison Birtle, consultant oncologist and honorary clinical professor, Lancashire Teaching Hospitals NHS Foundation Trust Patients, commented, “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. Unfortunately, until now, there have been limited treatment options available for this group of patients, which may affect not only their prognosis, but their well-being and quality of life. [The] authorisation of erdafitinib, a targeted therapy that has been shown to significantly improve overall and PFS for patients with FGFR3 alterations, will come as welcome news to eligible patients, and highlights the importance of integrating biomarker testing into the treatment pathway for people with urothelial cancer, so that genetic alterations such as FGFR3 can be detected as early as possible.”

Approximately 10,500 people in the UK are diagnosed with bladder cancer every year, which equates to 29 people per day. Around 20% of people with advanced or metastatic bladder cancer have FGFR3 alterations, which can drive the growth of cancer cells.