CHMP okays first therapy targeting FGFR3 alterations in bladder cancer


The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for Janssen-Cilag’s Balversa (erdafitinib).

It is recommended as a monotherapy to treat adult patients with unresectable or metastatic urothelial carcinoma, 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.

If it is approved, erdafitinib would become the first therapy targeting FGFR3 alterations in patients with metastatic urothelial carcinoma.

The recommendation is based on results from Cohort 1 of the Phase III THOR study, which showed a 36% reduction in the risk of death with erdafitinib versus chemotherapy.

“FGFR alterations are important oncogenic drivers in urothelial carcinoma and can be associated with adverse clinical outcomes,” said Yohann Loriot, Institut Gustave Roussy and University of Paris-Saclay, France. “As we aim to optimise treatment outcomes for patients, there is a significant unmet need for novel, targeted therapies that enable treatment decisions tailored according to a patient’s individual genetic and disease characteristics.”

Europe has one of the highest rates of bladder cancer in the world, with nearly 225,000 patients diagnosed in 2022, representing a 10% increase from 2020. The most common form of bladder cancer is UC, and up to 20% of patients with metastatic UC have FGFR alterations.

The results of the THOR study demonstrate median overall survival of over one year at the data cut-off, marking a significant increase as compared to those in the chemotherapy arm (12.1 months vs 7.8 months).

Treatment with erdafitinib also showed an improvement in median progression-free survival compared to chemotherapy of 5.6 months vs 2.7 months and overall response rate of 35.3% vs 8.5%.

Diana Spencer, Senior Digital Content Editor, DDW

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