Nearly 1,700 people are diagnosed with bladder cancer each year in Scotland, with almost all bladder cancers being urothelial cancers1
Up to one in five advanced bladder cancer patients have an FGFR3 genetic alteration, and face a poor prognosis2,3
10 urothelial cancer patients in Scotland are anticipated to receive this treatment per year1
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.3
Nearly 1,700 people are diagnosed with bladder cancer each year in Scotland, and almost all bladder cancers – more than 90 percent – are UCs.1,4,5 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.2 As a once-daily, oral FGFR3 kinase inhibitor, erdafitinib works by inhibiting the activity of FGFR alterations in cancer cells, thereby slowing tumour growth.4,6
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.3,5 The most common adverse reactions observed in the study included hyperphosphataemia (78.5 percent), diarrhoea (55.5 percent), and stomatitis (52.8 percent).4 Adverse reactions leading to treatment discontinuation occurred in 19.4 percent of patients.4
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.7
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.”
#ENDS#
Notes to Editors:
About erdafitinib
Erdafitinib is a once-daily, oral pan-fibroblast growth factor receptor 3 (FGFR3) tyrosine kinase inhibitor approved for the treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), harbouring susceptible FGFR3 genetic alterations 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 in the unresectable or metastatic treatment setting.4 It works by inhibiting the activity of FGFR3-expressing cancer cells, thereby stopping or slowing their growth.8
Important safety information
For a full list of side effects and information on dosage and administration, contraindications, special warnings and precautions when using erdafitinib, please refer to the Summary of Product Characteristics for further information.
About the THOR study4,5,9
THOR (NCT03390504) is a Phase 3 randomised, open-label, multicentre study evaluating the efficacy and safety of erdafitinib. All patients included in the study had metastatic or unresectable UC, with selected FGFR3 genetic alterations and showed disease progression during or after one or two prior lines of treatment. The study compared erdafitinib in two cohorts; erdafitinib versus standard of care chemotherapy (investigators choice of docetaxel or vinflunine) after at least one line of treatment including a PD-1 or PD-L1 inhibitor (Cohort 1); and erdafitinib compared to pembrolizumab after one prior treatment not containing a PD-1 or PD-L1 inhibitor (Cohort 2). The primary endpoint of the study was overall survival (OS), with secondary endpoints being progression-free survival (PFS), objective response rate (ORR), duration of response (DOR) and safety.
In June 2023, based on the recommendation of the independent data safety monitoring committee, the THOR study was stopped following review of efficacy and safety data of the study at interim analysis. All patients randomised to chemotherapy (docetaxel or vinflunine) were offered the opportunity to receive erdafitinib as crossover therapy. The results show median OS of over one year was achieved in patients receiving erdafitinib at the data cut-off, marking a significant improvement compared to those in the chemotherapy arm (12.1 months vs. 7.8 months; hazard ratio [HR], 0.64; 95 percent confidence interval [CI], 0.47 to 0.88; P=0.005). Treatment with erdafitinib also showed an improvement in median PFS compared to chemotherapy of 5.6 months versus 2.7 months (HR 0.58; 95 percent CI, 0.41 to 0.82; P=0.0002) and a confirmed ORR of 48 out of 136 patients (35.3 percent) versus 11 out of 130 patients (8.5 percent).
The most common adverse reactions include hyperphosphataemia (78.5 percent), diarrhoea (55.5 percent), and stomatitis (52.8 percent). Further common adverse reactions were dry mouth (39.9 percent), decreased appetite (31.7 percent) dry skin (28.0 percent), central serous retinopathy (28.0 percent), anaemia (28.2 percent), constipation (27.3 percent), dysgeusia (26.3 percent), palmar-plantar erythrodysaesthesia syndrome (PPES) (25.5 percent), alopecia (23.2 percent), asthenia (23.0 percent), alanine aminotransferase increased (21.7 percent), onycholysis (21.7 percent), fatigue (20.3 percent), nausea (18.6 percent), weight decreased (18.4 percent), aspartate aminotransferase increased (18.0 percent), dry eye (16.7 percent), nail discolouration (15.9 percent), vomiting (13.8 percent), blood creatinine increased (13.8 percent), hyponatraemia (13.4 percent), paronychia (12.5 percent), nail dystrophy (11.9 percent), onychomadesis (11.5 percent), epistaxis (10.6 percent) nail disorder (10.2 percent) and abdominal pain (10.0 percent). Most common Grade 3 or higher ADRs were stomatitis (10.6 percent), hyponatraemia (8.8 percent), palmar-plantar erythrodysaesthesia syndrome (7.9 percent), onycholysis (4.8 percent), diarrhoea (4.0 percent), hyperphosphataemia (2.9 percent), decreased appetite (2.5 percent), and nail dystrophy (2.5 percent). Grade 3 or 4 related TEAEs (47.6 percent vs 43.5 percent) and related serious adverse events (14.6 percent vs 10.5 percent) were reported more frequently for patients 65 years and older versus patients <65 years.
Adverse reactions leading to dose reduction occurred in 59.7 percent of patients. Stomatitis (15.4 percent), palmar-plantar erythrodysaesthesia syndrome (9.6 percent), onycholysis (7.3 percent) and hyperphosphataemia (5.2 percent) were the most common adverse events leading to dose reduction.
Adverse reactions leading to treatment discontinuation occurred in 19.4 percent of patients. Detachment of retinal pigment epithelium (1.7 percent) and stomatitis (1.5 percent) were the most common adverse events leading to treatment discontinuations.
About urothelial cancer
UC starts in the innermost lining of the bladder. Almost all bladder cancers – more than 90 percent – are UCs.5 Up to one in five patients (20 percent) diagnosed with advanced bladder cancer have a FGFR3 genetic alteration.2 FGFRs are a family of receptor tyrosine kinases that can be activated by genetic alterations in a variety of tumour types, and these alterations may lead to increased tumour cell growth and survival.10 FGFRs play a key role in regulating several biological processes, including tissue homeostasis, wound repair, and cancer.10 Fusions or mutations (also known as alterations) in the genes that control FGFR3 (known as FGFR1–4 alterations) may lead to the development and progression of certain cancers by increasing tumour cell growth.10 Patients with advanced UC, including FGFR3-driven tumours, face a poor prognosis and the need for innovative therapies remains high.11
About Johnson & Johnson
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Cautions Concerning Forward-Looking Statements
This press release contains “forward-looking statements” as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of erdafitinib. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialise, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC, Janssen Biotech, Inc., Janssen-Cilag Limited and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson’s Annual Report on Form 10-K for the fiscal year ended December 31, 2024, including in the sections captioned “Cautionary Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in Johnson & Johnson’s subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at www.sec.gov, www.jnj.com or on request from Johnson & Johnson. None of Janssen Research & Development, LLC, Janssen Biotech, Inc. Janssen-Cilag Limited nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.
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