J&J Innovative Medicine’s Balversa offers new hope for urothelial cancer patients with FGFR mutations

Johnson & Johnson (J&J) Innovative Medicine’s fibroblast growth factor receptor (FGFR) inhibitor Balversa (ingredient: erdafitinib) is gaining traction in the global market, including Korea, as a promising treatment option for urothelial cancer patients.

Balversa, a small molecule inhibitor targeting the FGFR, has shown significant potential in treating patients with locally advanced or metastatic urothelial cancer harboring FGFR2 or FGFR3 mutations.

At a press conference held at the Plaza Hotel on Wednesday to celebrate the drug's launch, experts shared their insights on the significance of Balversa in the treatment landscape.

Professor Kim In-ho of the Department of Medical Oncology at Seoul St. Mary's Hospital emphasized the importance of Balversa’s launch.

"With the five-year relative survival rate for metastatic urothelial cancer at just 11.7 percent, there was a significant unmet need,” Kim said. “Balversa, as an FGFR mutation-targeted therapy, offers improved survival rates and additional treatment opportunities for patients who have exhausted chemotherapy and immunotherapy options."

Balversa received approval from the Ministry of Food and Drug Safety in January 2022 for adult patients with locally advanced or metastatic urothelial cancer with FGFR2 or FGFR3 mutations. The approved indications include patients whose disease has progressed during or following at least one line of platinum-based chemotherapy, or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

However, following the approval of immunotherapy as first- and second-line treatment options for urothelial cancer, Balversa faced the challenge of demonstrating its efficacy in patients who had previously received these newer treatments.

J&J Innovative Medicine addressed this challenge through the phase 3 THOR clinical trial, which led to full approval from the U.S. Food and Drug Administration (FDA).

During the press conference, the latest clinical data was presented. The THOR trial results showed that Balversa reduced the risk of death by 36 percent compared to chemotherapy in patients who had received prior PD-1 or PD-L1 inhibitor therapy. The median overall survival was 12.1 months for the Balversa group versus 7.8 months for the chemotherapy group, a 55.13 percent increase.

Progression-free survival also more than doubled, with a median of 5.6 months for Balversa compared to 2.7 months for chemotherapy. Importantly, the survival benefits were consistent across various subgroups, and the safety profile was manageable.

"Looking at this study, despite being a phase 3 clinical trial, it clearly showed better results than previous treatments," Kim said. "Notably, these research results were not skewed towards specific patients but showed consistent results across subgroups."

Kim stressed that in terms of side effects, they were similar to previous studies, and if doctors keep this in mind, it can be adequately managed.

"Urothelial cancer requires diverse treatment options from early to late stages due to differences in mechanisms and approval conditions among available therapies,” Kim said. “Balversa's ability to improve survival rates and provide additional treatment opportunities for patients who have exhausted chemotherapy and immunotherapy options is truly significant."

Also speaking at the event, Professor Kim Tae-jung of the Department of Hospital Pathology at Yeouido St. Mary's Hospital emphasized the importance of rapid diagnosis of FGFR mutations in urothelial cancer when clinically necessary.

Among various cancer types, FGFR mutations are observed in about 20 percent of urothelial cancer patients, showing a particularly high frequency of mutation detection. FGFR plays a crucial role in signaling pathways that regulate cell growth, differentiation, survival, and migration."

“The National Comprehensive Cancer Network (NCCN) guidelines recommend considering molecular/genomic testing for certain urothelial cancer patients, especially those with a tumor stage of T3 or higher,” he said.