Johnson & Johnson Announces China Approval of BALVERSA® (Erdafitinib) for FGFR3-Altered Locally Advanced or Metastatic Urothelial Carcinoma

On January 13, 2025, Johnson & Johnson announced that its innovative therapeutic drug Balversa (erdafitinib tablets) has been officially approved by the National Medical Products Administration for the treatment of adult patients with surgically unresectable locally advanced or metastatic urothelial carcinoma (UC) who carry susceptible FGFR3 gene alterations and have experienced disease progression during or after at least one prior line of therapy containing anti-PD-1 or anti-PD-L1 [2].

Erdafitinib tablets are a novel targeted therapy drug that can significantly improve the overall survival and progression-free survival of patients with FGFR3 gene mutations [2], providing a new treatment option for patients with limited prior treatment choices. Notably, for patients with advanced bladder cancer, the guidelines released by the European Society for Medical Oncology in 2024 and the European Association of Urology in 2024 both specifically recommend that molecular/genetic testing should be conducted as soon as possible after diagnosis to facilitate more precise treatment decisions and avoid delays that could impact subsequent treatment [1,3,4].

Cherry Huang, President of Johnson & Johnson Innovative Pharmaceuticals China, stated: "Bocoi demonstrates the significant benefits and broad application prospects that targeted therapy brings to patients with advanced bladder cancer, highlighting the importance of FGFR gene testing in the diagnosis and treatment of metastatic urothelial carcinoma. As an important milestone in this field, this approval showcases Johnson & Johnson's long-term commitment to advancing precision treatment in oncology and improving patient survival benefits. In the future, we will continue to focus on areas with significant unmet medical needs, leading the future of medicine with science, fully promoting the whole lifecycle management of cancer, and striving to make cancer a controllable and curable chronic disease."
Bladder cancer is one of the top ten most common tumors in Chinese men [5], with the most common type being urothelial carcinoma [6], accounting for approximately 90% [2]. About 20% of patients with metastatic urothelial carcinoma carry FGFR gene alterations [7]. The prognosis for patients with metastatic urothelial carcinoma is generally poor, with a five-year survival rate of only 5% for those with advanced metastasis [8].

This approval of Bokē is based on a Phase III clinical trial, the THOR (BLC3001) study [2,9]. The primary endpoint, overall survival (OS), showed that patients treated with Erdafitinib had a statistically significant improvement in OS compared to chemotherapy. Erdafitinib significantly extended OS (median OS was 12.1 months versus 7.8 months in the chemotherapy group (HR=0.64; 95% CI, 0.47-0.88; p=0.0050)). This indicates that patients receiving Erdafitinib treatment had a 36% reduction in the risk of death compared to the chemotherapy group. Patients treated with Erdafitinib also showed improvement in median progression-free survival (PFS), which was 5.6 months (95% CI, 4.4 - 5.7) compared to 2.7 months (95% CI, 1.8 - 3.7) in the chemotherapy group. Additionally, the objective response rate (ORR) improved, with 35.3% in the Erdafitinib group versus 8.5% in the chemotherapy group (relative benefit, 4.16; 95% CI, 2.27-7.64) [2,9].

Studies show that the safety of Erdafitinib tablets is generally controllable. The most common adverse reactions (≥20%) include hyperphosphatemia, diarrhea, oral mucositis, dry mouth, decreased appetite, dry skin, anemia, constipation, dysgeusia, palmar-plantar erythrodysesthesia syndrome (PPES), alopecia, increased alanine aminotransferase, nail detachment, and weight loss [2,9].
References:
[1] The 2024 European Association of Urology Guidelines on Muscle-invasive and Metastatic Bladder Cancer.
[2] BALVERSA China Prescribing Information, December 2024.
[3] Powles T et al. ESMO Clinical Practice Guideline interim update on first-line therapy in advanced urothelial carcinoma. Annals of Oncology 2024;35(6): 485-490.
[4] Powles T, et al. Bladder cancer: ESMO clinical practice guideline for diagnosis, treatment and follow-up. Ann Oncol 2022;33(3):244–258.
[5] Chinese Urological Oncology Collaboration Group of Cancer Hospitals. Expert Consensus on Early Diagnosis and Treatment of Bladder Cancer (2024 Edition) [J]. China Cancer, 2024, 34(6): 607-618.
[6] Fernandez E, et al. Prognostic value and clinical significance of FGFR genomic alterations (GAs) in metastatic urothelial cancer patients. J Clin Med 2022;11(15):4483.
[7] Cancer.gov. Bladder Cancer Prognosis and Survival Rates – NCI (2023). Available at: https://www.cancer.gov/types/bladder/survival. Accessed August 2024.
[8] Montazeri K & Bellmunt J. Erdafitinib for the treatment of metastatic bladder cancer. Expert Review of Clinical Pharmacology. 2020;13(1):1-6..
[9] 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.