Erdafitinib ( Balversa ) is a pan-FGFR kinase inhibitor for treatment of adults with metastatic urothelial carcinoma ( mUC ) with susceptible FGFR3/2 alterations and who progressed on 1 line or more of prior Platinum-based chemotherapy.
Approval was based on data from the primary analysis of the pivotal BLC2001 trial.
Researchers have reported long-term efficacy and safety data from the 8 mg/d continuous dose regimen in BLC2001.
BLC2001 is a global, open-label, phase 2 trial of patients with measurable metastatic urothelial carcinoma with prespecified FGFR alterations, ECOG 0-2, and progression during / following 1 line or more of prior chemotherapy or less than or equal to 12 months of (neo)adjuvant chemotherapy, or were Cisplatin ineligible, chemotherapy naïve.
The optimal schedule of Erdafitinib determined in the initial part of the study was 8 mg/d continuous Erdafitinib in 28-d cycles with uptitration to 9 mg/d ( Erdafitinib 8 mg UpT ) if a protocol-defined target serum phosphate level was not reached and if no significant treatment-related adverse events ( TRAEs ) occurred.
Primary end point was the confirmed objective response rate ( ORR=% complete response + % partial response ).
Key secondary end points were progression-free survival ( PFS ), duration of response ( DOR ) and overall survival ( OS ).
Median follow-up for 101 patients treated with Erdafitinib 8 mg UpT was approximately 24 months.
Confirmed ORR was 40%. Median DOR was 5.98 months; 31% of responders had DOR greater than or equal to 1 year.
Median PFS was 5.52 months, median OS was 11.3 months. 12-months and 24-months survival rates were 49% and 31%, respectively.
Median treatment duration was 5.4 months.
The Erdafitinib safety profile was consistent with the primary analysis. No new TRAEs were seen with longer follow-up.
Central serous retinopathy ( CSR ) events occurred in 27% ( 27/101 ) of patients; 85% ( 23/27 ) were grade 1 or 2; dosage was reduced in 13 patients, interrupted for 8, and discontinued for 3.
On the data cut-off date, 63% ( 17/27 ) had resolved; 60% ( 6/10 ) of ongoing CSR events were grade 1.
There were no treatment-related deaths.
In conclusion, with a median follow-up of 2 years, Erdafitinib in mUC + FGFR alterations showed a manageable safety profile and consistent efficacy, with median overall survival of 11.3 months.
31% had a DOR greater than or equal to 12 months and 31% were alive at 24 months. ( Xagena )
Source: American Society of Clinical Oncology ( ASCO ) Virtual Meeting, 2020