NICE recommends BALVERSA®▼ (erdafitinib), the first and only treatment in the UK for metastatic or unresectable fibroblast growth factor receptor (FGFR3)-altered urothelial cancer (UC) for patients who have previously received at least one line of therapy containing a PD-1 or PD-L1 inhibitor

Over 19,000 people are diagnosed with bladder cancer each year in the UK, with almost all bladder cancers being urothelial cancers^[1],[2]

Up to one in five advanced bladder cancer patients have an FGFR3 genetic alteration, and face a poor prognosis^[3],[4]

Erdafitinib is the first and only oral bladder cancer therapy that specifically targets FGFR3 alterations^[5]

High Wycombe, UK (10 April 2025) – Johnson & Johnson has announced that the National Institute for Health and Care Excellence (NICE) has recommended BALVERSA^®▼ (erdafitinib), for the treatment of eligible adults with unresectable or metastatic urothelial cancer (UC) which is harbouring susceptible fibroblast growth factor receptor (FGFR3) genetic alterations, and 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.⁴

Almost all bladder cancers – more than 90 percent – are UCs.² Approximately 19,000 people in the UK are diagnosed with bladder cancer every year, and up to one in five patients (20 percent) diagnosed with metastatic or unresectable bladder cancer have an FGFR3 genetic alteration, which can drive the growth of cancer cells.^1,3 As a once-daily, oral FGFR3 kinase inhibitor, erdafitinib works by inhibiting the activity of FGFR alterations in cancer cells, thereby slowing tumour growth.^[6],[7],8

Jeannie Rigby, Chief Executive of Action Bladder Cancer UK, commented, “Erdafitnib represents a new area of treatment for bladder cancer. Bladder cancer has been badly neglected in terms of new treatments, and there are few current treatments available – which has had a direct impact on poorer outcomes for those with bladder cancer. It’s time that bladder cancer patients had some kind of new treatment available – we hope this is just the start of new hope for those with bladder cancer and their families.”

NICE primarily based its recommendation on data from the THOR study, which has shown erdafitinib to increase overall survival from 7.8 months to 12.1 months compared to chemotherapy in the second-line setting.⁷ The most common adverse reactions observed in the study included hyperphosphataemia (78.5 percent), diarrhoea (55.5 percent), and stomatitis (52.8 percent).⁶ Adverse reactions leading to treatment discontinuation occurred in 19.4 percent of patients.⁶

“People with advanced or metastatic bladder cancer tend to face a poor prognosis, and – until now – have had no targeted therapies available to them on the NHS.” said the British Uro-oncology Group, representing clinical and medical oncologists specialising in urology. “Today’s recommendation marks a step-change improvement in the management of urothelial cancer. As both a targeted and oral treatment, this potentially life-extending precision medicine option is a welcome advance for this disease.”

“We are delighted to have reached this outcome for people living with advanced urothelial cancer,” said Dr. John Fleming, UK Country Medical Director, Johnson & Johnson Innovative Medicine. “NICE’s swift recommendation of erdafitinib is testament to what the therapy represents for the urothelial cancer community, and to the commitment and hard work of everyone involved in the appraisal process. At J&J, we know how important it is for cancer patients to have access to therapies that can target the specific characteristics of their disease. We look forward to seeing the benefits that access to a targeted medicine on the NHS may bring to patients with FGFR3-positive urothelial cancer, and we hope to see an increase in genetic testing for urothelial cancer patients to enable the quick identification of genetic alterations, such as FGFR3.”

[1] Fight Bladder Cancer. What is bladder cancer? Available at https://www.fightbladdercancer.co.uk/get-help/what-bladder-cancer/. Last accessed April 2025.

[2] Leslie SW, Soon-Sutton TL, Aeddula NR. Bladder Cancer. In: StatPearls. Treasure Island (FL): StatPearls Publishing; 15 August 2024.

[3] Xiao JF, Caliri AW, Duex JE, Theodorescu D. Targetable Pathways in Advanced Bladder Cancer: FGFR Signaling. Cancers (Basel). 2021;13(19):4891.

[4] NICE. Erdafitinib for treating metastatic or unresectable FGFR-altered urothelial cancer. Available at https://www.nice.org.uk/guidance/indevelopment/gid-ta10252. Last accessed April 2025.

[5] Ascione CM, et al. Role of FGFR3 in bladder cancer: Treatment landscape and future challenges. Cancer Treat Rev. 2023;115:102530. 

[6] BALVERSA® (erdafitinib). Summary of Products Characteristics (3 mg, 4 mg and 5 mg tablets). Available at https://www.medicines.org.uk/emc/search?q=balversa. Last accessed 8 April 2025.

[7] Loriot Y, Matsubara N, Park SH et al. Erdafitinib or Chemotherapy in Advanced or Metastatic Urothelial Carcinoma. N Engl J Med. 2023;389(21):1961-1971.