10,500 people in the UK are diagnosed with bladder cancer every year1
Around 20 percent of people with advanced or metastatic bladder cancer have fibroblast growth factor receptor 3 (FGFR3) alterations, which can drive the growth of cancer cells2
Erdafitinib is the first and only bladder cancer therapy that targets FGFR3 alterations and has been shown to increase overall survival from 7.8 months to 12.1 months (with a 95 percent confidence interval) compared to chemotherapy3,4
High Wycombe, UK (06 November 2024) – Johnson & Johnson has announced that the Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for BALVERSA®▼ (erdafitinib) as a monotherapy for the treatment of adults with unresectable or metastatic urothelial carcinoma (UC), the most common form of bladder cancer.5,6 Specifically, the indication covers eligible patients harbouring susceptible fibroblast growth factor receptor 3 (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.5
Approximately 10,500 people in the UK are diagnosed with bladder cancer every year, which equates to 29 people per day.1 Around 20 percent of people with advanced or metastatic bladder cancer have FGFR3 alterations, which can drive the growth of cancer cells.2
“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,” said Professor Alison Birtle, Consultant Oncologist and Honorary Clinical Professor, Lancashire Teaching Hospitals NHS Foundation Trust. “Unfortunately, until now, there have been limited treatment options available for this group of patients, which may affect not only their prognosis, but their wellbeing and quality of life. Today’s authorisation of erdafitinib, a targeted therapy that has been shown to significantly improve overall and progression-free survival 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.”
Erdafitinib is a once-daily, oral FGFR kinase inhibitor, which works by inhibiting the activity of FGFR3 alterations in cancer cells and has been shown to extend overall survival compared to chemotherapy in the second-line setting.3
Today’s MHRA authorisation is based on results from Cohort 1 of the Phase 3 THOR study, a randomised, open-label, multicentre study which evaluated the efficacy and safety of erdafitinib (n=136) versus chemotherapy (n=130) in patients with advanced or metastatic UC with select FGFR 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.3,7 The primary endpoint of the study was overall survival (OS), with secondary endpoints being progression-free survival (PFS), objective response rate (ORR) and duration of response (DOR).3
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.3 All patients randomised to chemotherapy (docetaxel or vinflunine) were offered the opportunity to receive erdafitinib as crossover therapy.3 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], 0.44 to 0.93; P=0.005).5 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).5
The most common adverse reactions include hyperphosphataemia (78.5 percent), diarrhoea (55.5 percent), and stomatitis (52.8 percent).* Adverse reactions leading to treatment discontinuation occurred in 19.4 percent of patients.5
“We are delighted that the MHRA has recognised the value that erdafitinib could bring to eligible patients with metastatic urothelial cancer,” said Dr. John Fleming, Country Medical Director, Johnson & Johnson Innovative Medicine UK. “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.”
#ENDS#
Notes to Editors:
*Further common adverse reactions were dry mouth (39.9 percent), decreased appetite (31.7 percent) dry skin (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), alanine aminotransferase increased (21.7 percent), onycholysis (21.7 percent), nausea (18.6 percent), weight decreased (21.7 percent), aspartate aminotransferase increased (18 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) and nail disorder (10.2 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.5
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.5
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.5
About the THOR study3,8
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 FGFR 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 trial consisted of a screening step, a treatment phase (from randomisation until disease progression, intolerable toxicity, withdrawal of consent or decision by investigator to discontinue treatment) and a post-treatment follow-up (from end-of-treatment to participants death, withdrawal of consent, lost to follow-up, study completion for the respective cohort, whichever comes first). The primary endpoint of the study was OS, with secondary endpoints being PFS, ORR and DOR.
About erdafitinib
Erdafitinib is a once-daily, oral pan-fibroblast growth factor receptor (FGFR) 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 PD-1 or PD-L1 inhibitor in the unresectable or metastatic treatment setting.5 It works by inhibiting the activity of FGFR-expressing cancer cells, thereby stopping or slowing their growth.9
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 urothelial carcinoma
Urothelial carcinoma (UC) starts in the innermost lining of the bladder.6 Almost all bladder cancers – more than 90 percent – are UCs.6 Up to one in five patients (20 percent) diagnosed with metastatic UC have a fibroblast growth factor receptor (FGFR) 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 several biological processes, including tissue repair, inflammatory response and metabolism.10 Fusions or mutations (also known as alterations) in the genes that control FGFR (known as FGFR1–4 alterations) may lead to the development and progression of certain cancers by increasing tumour cell growth and survival.11 Patients with advanced UC, including FGFR-driven tumours, face a poor prognosis and the need for innovative therapies remains high.12
About Johnson & Johnson
Janssen, the Pharmaceutical Companies of Johnson & Johnson, has become Johnson & Johnson Innovative Medicine. The Company is updating its brand and uniting both its pharmaceutical and MedTech segments under the Johnson & Johnson brand name.
At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity.
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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, 2023, 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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