SMC approval of BALVERSA®▼ (erdafitinib) helps pave the way for first licensed bladder cancer therapy that specifically targets fibroblast growth factor receptor (FGFR3) alterations

Nearly 1,700 people are diagnosed with bladder cancer each year in Scotland [i]

Up to one in five advanced bladder cancer patients have an FGFR3 genetic alteration [ii] [iii]

10 urothelial cancer patients in Scotland are anticipated to receive this treatment per year [i]

High Wycombe, UK (12 May 2025) – Johnson & Johnson is delighted to announce that BALVERSA®▼ (erdafitinib) is accepted for use within NHS Scotland 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.[iii]  

Nearly 1,700 people are diagnosed with bladder cancer each year in Scotland, and almost all bladder cancers – more than 90 percent – are UCs.[i], [iv] [v] Up to one in five patients (20 percent) diagnosed with metastatic bladder cancer have an FGFR3 genetic alteration, which can drive the growth of cancer cells.[ii] As a once-daily, oral FGFR3 kinase inhibitor, erdafitinib works by inhibiting the activity of FGFR alterations in cancer cells, thereby slowing tumour growth. [iv] [vi]

The SMC 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.iii,v The most common adverse reactions observed in the study included hyperphosphataemia (78.5 percent), diarrhoea (55.5 percent), and stomatitis (52.8 percent).[iv] Adverse reactions leading to treatment discontinuation occurred in 19.4 percent of patients.[iv]

The SMC’s acceptance of erdafitinib follows the National Institute for Health and Care Excellence (NICE) approval for the same indication received last month, meaning that eligible patients across Great Britain can now access erdafitinib. [viii]

Prof. Rob Jones, Professor of Clinical Cancer Research at University of Glasgow, said: “This is good news for patients with incurable forms of bladder cancer, as it adds an additional line of life-prolonging treatment for some patients. Moreover, because the treatment is only suitable for patients with specific genetic changes, we can accurately select patients where the treatment is more likely to work. This means we can spare the other patients from the side effects of a treatment which would be ineffective. This will be the first such ‘precision medicine’ targeted therapy for patients with advanced bladder cancer harbouring susceptible FGFR3 genetic alterations.”

“The SMC’s acceptance of erdafitinib means that eligible urothelial cancer patients from across the UK can now access this first in class treatment option,” said Dr. John Fleming, UK Country Medical Director, Johnson & Johnson Innovative Medicine. “This news marks a significant step forward for the bladder cancer community and reaffirms our commitment to delivering innovative therapies that can potentially help extend patients’ lives.”

• [i] NHS Scotland. Bladder Cancer. Available at https://www.getcheckedearly.org/bladder-cancer. Last accessed May 2025.
• [ii] Xiao JF, Caliri AW, Duex JE, Theodorescu D. Targetable Pathways in Advanced Bladder Cancer: FGFR Signaling. Cancers (Basel). 2021;13(19):4891.
• [iii] SMC. Erdafitinib (Balversa). Available at https://scottishmedicines.org.uk/medicines-advice/erdafitinib-balversa-full-smc2738/. Last accessed May 2025.
• [iv] 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 01 May 2025.
• [v] Leslie SW, Soon-Sutton TL, Aeddula NR. Bladder Cancer. In: StatPearls. Treasure Island (FL): StatPearls Publishing; 15 August 2024.
• [vi] 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.
• [vii] NICE. Erdafitinib for treating metastatic or unresectable FGFR-altered urothelial cancer. Available at https://www.nice.org.uk/guidance/indevelopment/gid-ta10252. Last accessed May 2025.
• [viii] Roubal K, Myint ZW, Kolesar JM. Erdafitinib: A novel therapy for FGFR-mutated urothelial cancer. Am J Health Syst Pharm. 2020;77(5):346-351.
• [ix] Siefker-Radtke AO, Matsubara N, Park SH et al. Erdafitinib versus pembrolizumab in pretreated patients with advanced or metastatic urothelial cancer with select FGFR alterations: cohort 2 of the randomized phase III THOR trial. Ann Oncol. 2024;35(1):107-117.
• [x]  Presta M et al. Fibroblast growth factors (FGFs) in cancer: FGF traps as a new therapeutic approach. Pharmacol Ther. 2017;179:171-187.
• [xi] Loriot Y, Necchi A, Park SH et al. Erdafitinib in Locally Advanced or Metastatic Urothelial Carcinoma. N Engl J Med. 2019;381(4):338-348.