Janssen's Erdafitinib (Bokew) Approved in China as First Targeted Therapy for Locally Advanced or Metastatic Urothelial Carcinoma with FGFR3 Alterations

China Finance and Economics News, January 13th: Johnson & Johnson announced today 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.

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, offering a new treatment option for patients with limited prior treatment choices. Notably, for patients with advanced bladder cancer, guidelines released by the European Society for Medical Oncology in 2024 and the European Association of Urology in 2024 both specifically emphasize that molecular/genetic testing should be conducted as soon as possible after diagnosis to facilitate more precise treatment decisions, prevent delays in the condition, and avoid impacting subsequent treatments.

Cherry Huang, President of Johnson & Johnson Innovative Medicine China, stated that Bocoe demonstrates the significant benefits and broad application prospects of targeted therapy for 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 underscores Johnson & Johnson's long-term commitment to advancing precision medicine 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."

Data show that bladder cancer is one of the top ten most common tumors in Chinese men, with the most common type being urothelial carcinoma, accounting for about 90%. Approximately 20% of patients with metastatic urothelial carcinoma carry FGFR gene mutations. 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.

The approval of Bocoe this time is based on a Phase III clinical trial, the THOR (BLC3001) study. The primary endpoint, overall survival (OS), showed that compared with chemotherapy, patients receiving Erdafitinib had a statistically significant improvement in OS. Erdafitinib significantly prolonged 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. In addition, the objective response rate (ORR) was also 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).

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.