On January 19, 2024, erdafitinib (Balversa) was approved for patients with locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 genetic alterations, as determined by a U.S. Food & Drug Administration–approved companion diagnostic test, whose disease has progressed on or after at least one line of prior systemic therapy.¹ Erdafitinib is not recommended for patients who are eligible for and have not received prior PD-1 or PD-L1 inhibitor therapy. This approval amends the April 2019 accelerated approval for patients with susceptible FGFR3 or FGFR2 alterations.

Supporting Efficacy Data

Approval was based on the open-label Study BLC3001 (ClinicalTrials.gov identifier NCT03390504) Cohort 1. In this cohort, 266 patients with selected FGFR3 alterations who had received one or two prior systemic treatments, including a PD-1 or PD-L1 inhibitor, were randomly assigned to receive oral erdafitinib at 8 mg once daily with titration up to 9 mg (n = 136) or chemotherapy (docetaxel at 75 mg/m² or vinflunine at 320 mg/m² once every 3 weeks) until disease progression or unacceptable toxicity.

Median overall survival was 12.1 months (95% confidence interval [CI] = 10.3–16.4 months) in the erdafitinib group vs 7.8 months (95% CI = 6.5–11.1 months) in the chemotherapy group (hazard ratio [HR] = 0.64, 95% CI = 0.47–0.88, P = .0050). The erdafitinib group also had superior median progression-free survival (5.6 months vs 2.7 months; HR = 0.58, 95% CI = 0.44–0.78, P = .0002) and objective response rate (35.3% vs 8.5%, P < .001).

How It Is Used

The recommended erdafitinib dose is 8 mg once daily, with a dose increase to 9 mg once daily based on tolerability at 14 to 21 days. Treatment should continue until disease progression or unacceptable toxicity.

Safety Profile

In Study BLC3001 Cohort 1, the most common adverse events of any grade in the erdafitinib group were nail disorders (70% vs 5% in chemotherapy group), diarrhea (63% vs 17%), stomatitis (56% vs 18%), dry mouth (39% vs 4%), palmar-plantar erythrodysesthesia syndrome (30% vs 1%), and dysgeusia (30% vs 7%), as well as fatigue, dry skin, constipation, decreased appetite, alopecia, dry eye, and increased and decreased weight. The most common grade 3 or 4 laboratory abnormalities were decreased hemoglobin (12%), decreased phosphate (8%), and decreased calcium (8%).

Erdafitinib has warnings/precautions for ocular disorders, hyperphosphatemia, and embryofetal toxicity. Patients should be advised not to breastfeed while receiving erdafitinib. 

REFERENCE

1. Balversa (erdafitinib) tablets prescribing information, Janssen Pharmaceutical, January 2024. Available at www.janssenlabels.com/package-insert/product-monograph/prescribing-information/BALVERSA-pi.pdf. Accessed February 1, 2024.