Bladder cancer drug Balversa offers new treatment option

First targeted therapy approved by the US FDA and in Korea for urothelial cancer

Phase III study shows a 36% reduction in risk of death and improved PFS and OS compared to chemotherapy

Experts emphasize the importance of early detection of FGFR mutations

The introduction of the targeted therapy Balversa (erdafitinib) in urothelial carcinoma has attracted attention for its potential to address unmet needs.

 

In particular, the emergence of FGFR inhibitors has highlighted the importance of genetic mutation diagnostics to quickly detect the presence of such mutations.

 

(from the left) Professor Tae-Jung Kim, Professor Inho Kim, BU director Yeon-Hee Kim

Inho Kim, professor of Oncology at Seoul St.

 

Mary's Hospital, and Tae-Jung Kim, professor of pathology at Yeouido St.

 

Mary's Hospital, emphasized the importance of diagnosing FGFR mutations at a press conference held by Janssen Korea on the 16th, which was held to celebrate the launch of Balversa.

 

Balversa was approved by the Ministry of Food and Drug Safety in January 2022.

 

However, it is still not reimbursed in Korea.

 

Specifically, the drug is indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC) with FGFR2 or FGFR3 genetic alterations whose disease has progressed on or after at least one line of prior systemic therapy, which includes platinum-based chemotherapy, or whose disease has progressed within 12 months of neoadjuvant or adjuvant treatment with platinum-based chemotherapy.

 

However, the approval of PD-1 and PD-L1-directed immuno-oncology agents in the first- and second-line settings that followed Balversa’s approval led to the need for Balversa to demonstrate efficacy in patients who previously received these agents.

 

The situation was addressed with the publication of Balversa’s Phase III THOR trial study, which demonstrated a prolonged overall survival (OS) benefit with Balversa over chemotherapy in patients with metastatic urothelial carcinoma with FGFR3/2 gene alterations whose disease progressed after first-line treatment with immuno-oncology agents.

 

In the study, Balversa prolonged overall survival (OS) compared with chemotherapy in patients with metastatic urothelial carcinoma.

 

Results showed that over a median follow-up of 15.9 months, the mOS was 12.1 months in the Balversa arm, reducing the risk of death by 36% compared with the 7.8 months in the chemotherapy arm.

 

Based on these findings, the U.S.

 

Food and Drug Administration granted Balversa formal approval in January, but with a more restricted indication than originally approved.

 

“Bladder cancer is most commonly diagnosed in those in their 60s and older, with frequent recurrences and metastases, so it is important to prevent metastases or treat recurrences and metastases early,” said Professor In-ho Kim.

 

“There is a significant unmet therapeutic need, especially for patients with distant metastases, where the 5-year relative survival rate is only 11.7%.” ‘Balversa is the first targeted therapy for bladder cancer, which is significant because it improves survival in patients who have exhausted both chemotherapy and immuno-oncology options and provides an opportunity for further treatment,’ added Professor Kim.

 

The second speaker, Professor Tae-Jung Kim, emphasized the importance of early diagnosis of FGFR mutations in bladder cancer patients.

 

‘FGFRs play a significant role in signaling pathways that regulate cell growth, differentiation, survival and migration,’ said Professor Kim.

 

’FGFR mutations are found in a variety of cancers, but they are particularly common in bladder cancer, where they are observed in approximately 20% of patients.’ ‘The use of mutation-specific targeted therapies may help stop cancer proliferation and progression or improve the effectiveness of other treatments,’ he added.

 

’The NCCN guidelines also consider or recommend molecular/genomic testing for genetic mutations in some patients, such as those with bladder cancer tumor invasion grade IIIB or higher.’ In other words, for patients with bladder cancer who are in the chemotherapy phase, the guidelines recommend testing for genetic mutations in the early stages of treatment strategy planning.

 

“We are pleased to be able to offer a new treatment option in FGFR-mutated urothelial cancer with Balversa.

 

We plan to communicate the clinical benefits and emphasize the importance of mutation diagnosis so that more patients can benefit from Balversa treatment.”