Efficacy of erdafitinib in bladder cancer sustained with long-term follow-up

According to the results published in the Lancet Oncology.1

At a median follow-up of 24 months, the objective response rate with erdafitinib was 40%, comprising a complete response rate of 4.0% and a partial response rate of 36.0%. No new safety signal emerged with extended follow-up compared to safety data from the primary analysis.

“With longer follow-up, treatment with the selected treatment regimen of erdafitinib showed consistent activity and a manageable safety profile in patients with locally advanced or metastatic and pre-specified urothelial carcinoma. FGFR alterations, ”lead study author Arlene O. Siefker-Radtke, MD, professor in the Department of Genitourinary Medical Oncology, Division of Cancer Medicine, University of Texas MD Anderson Cancer Center, and co-authors wrote in the conclusion of their study.

Based on the main results of BLC2001, the FDA granted accelerated approval of erdafitinib in April 2019 for the treatment of adult patients with locally advanced or metastatic bladder cancer with a FGFR3 Where FGFR2 deterioration that has progressed with platinum-based chemotherapy.

Updated results from the multicenter, open-label, single-arm BLC2001 trial (NCT02365597) included data for 101 evaluable patients with metastatic or surgically unresectable urothelial cancer with FGFR genomic alterations. As confirmed by a central laboratory, patients had FGFR3 genetic mutations (R248C, S249C, G370C, Y373C) or FGFR gene fusions (FGFR3-TACC3, FGFR3-BAIAP2L1, FGFR2-BICC1, FGFR2-CASP7).

The median age of the patients was 67 years (range, 61-73). Ninety-three percent of patients had an ECOG performance index of 0 or 1 and 77% had visceral metastases. Only 10 patients had not received previous treatment. The remaining patients had received 1 (n = 48), 2 (n = 28) or ≥ 3 (n = 15) lines of treatment. Twelve percent of patients were chemotherapy-naïve, with 88% having progressed or relapsed after receiving chemotherapy. Twenty-four percent of patients had previously had immunotherapy.

The initial dose of erdafitinib was 8 mg once daily. Patients whose serum phosphate levels were below the target of 5.5 mg / dL between days 14 and 17 had their dose increased to 9 mg once daily. Patients were treated until unacceptable toxicity or disease progression.

The median duration of response was 6.0 months (95% CI, 4.2-7.5). Thirty-one (31%) of 101 patients achieved a response that lasted at least 1 year. Beyond the 40 (40%) responding patients, an additional 41 out of 101 patients had stable disease, resulting in an overall disease control rate of 80%.

Grade 3/4 all-cause treatment-emergent adverse events (TREs) occurred in 71% of 101 evaluable patients. The most frequently reported grade 3/4 all-cause TEEs were stomatitis (14%) and hyponatremia (11%). No treatment-related deaths occurred.

Summarizing the implications of these long-term results, Siefker-Radtke et al wrote: “The long-term follow-up of this study confirms the benefit of erdafitinib, an FGFR inhibitor, for the treatment of cancer patients. locally advanced or metastatic urothelial. and specific

Changes to the FGFR. Further research, in a randomized, controlled phase 3 study in patients with advanced urothelial cancer, is underway to evaluate erdafitinib as second-line monotherapy versus a PD-1 inhibitor or an chemotherapy. Another study is underway to assess erdafitinib in combination with a PD-1 inhibitor (cetrelimab) in the first-line treatment of patients with metastatic urothelial carcinoma ineligible for cisplatin. “


1. Siefker-Radtke AO, Necchi A, Park SH, et al. Efficacy and safety of erdafitinib in patients with locally advanced or metastatic urothelial carcinoma: long-term follow-up of a phase 2 study [published online January 11, 2022]. Lancet Oncol. doi: 10.1016 / S1470-2045 (21) 00660-4

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