On April 12, 2019, the U.S. Food and Drug Administration (USFDA) granted accelerated approval to Balversa (erdafitinib), a treatment for adult patients with locally advanced or metastatic bladder cancer that has a type of susceptible genetic alteration known as (fibroblast growth factor receptor) FGFR3 or FGFR2, and that has progressed during or following prior platinum-containing chemotherapy1. This approval represents the first personalized treatment targeting susceptible FGFR genetic alterations for patients with metastatic bladder cancer.

The approval followed by the efficacy study of Balversa included 87 patients with locally advanced or metastatic bladder cancer, with FGFR3 or FGFR2 genetic alterations, that had progressed following treatment with chemotherapy. The overall response rate in these patients was 32.2%, with 2.3% having a complete response and almost 30% having a partial response. The response lasted for an average of approximately five-and-a-half months. About a quarter of patients in the study were previously treated with anti PD-L1/PD-1 therapy, which is a standard treatment for patients with locally advanced or metastatic bladder cancer. Responses to Balversa were seen in patients who had previously not responded to anti PD-L1/PD-1 therapy.

Balversa received an Accelerated Approval and was also granted Breakthrough Therapy designation. The FDA granted the approval of Balversa to Janssen Pharmaceutical.

The FDA also approved the therascreen FGFR RGQ RT-PCR Kit, which is the first PCR-based companion diagnostic approved to detect FGFR alterations developed by QIAGEN Manchester, Ltd., for use as a companion diagnostic with Balversa for this therapeutic indication.

About Urothelial Carcinoma

Urothelial carcinoma is also known as transitional cell carcinoma, the most common type of bladder cancer. Bladder cancers are associated with genetic mutations that are present in the patient's bladder or entire urothelium (the lining of the lower urinary tract). Bladder cancer is the sixth most common cancer in the United States. Fibroblast growth factor (FGFR) alterations are present in approximately one in five patients with recurrent and refractory bladder cancer.

About Balversa (erdafitinib)2

BALVERSA (erdafitinib) is a once-daily, oral FGFR kinase inhibitor indicated for the treatment of adults with locally advanced or metastatic urothelial carcinoma (mUC) which has susceptible FGFR3 or FGFR2 genetic alterations and who have progressed during or following at least one line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.

Note - The approval of BALVERSA for said indication is under accelerated approval based on tumor response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. In US, BALVERSA will be commercially available through the single-source specialty pharmacy provider US Bioservices. Janssen entered into an exclusive worldwide license and collaboration agreement with Astex Pharmaceuticals to develop and commercialize BALVERSA.

Footnotes

1. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm635906.htm

2. https://www.janssen.com/balversa-erdafitinib-receives-us-fda-approval-treatment-patients-locally-advanced-or-metastatic

The content of this article is intended to provide a general guide to the subject matter. Specialist advice should be sought about your specific circumstances.