FDA Approves Erdafitinib for Locally Advanced or Metastatic Urothelial Carcinoma With FGFR3 Mutations


Erdafitinib was approved for the treatment of adults with locally advanced or metastatic urothelial carcinoma, which amends a previously approved indication of the drug.

FDA Approves Erdafitinib for Locally Advanced or Metastatic Urothelial Carcinoma With FGFR3 Mutations

FDA Approves Erdafitinib for Locally Advanced or Metastatic Urothelial Carcinoma With FGFR3 Mutations

The FDA has approved erdafitinib (Balversa) for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 genetic mutations whose disease has progressed on or after previously receiving one line of systemic therapy.

Of note, treatment with erdafitinib is not recommended for patients who are eligible for and have not received previous PD-1 or PD-L1 inhibitor therapy, according to a notice from the FDA.

This current FDA approval amends a previously granted indication for erdafitinib via accelerated approval for patients with metastatic urothelial carcinoma with susceptible FGFR2 or FGFR3 alterations after previous platinum-containing chemotherapy.

The approval was based on findings from the Study BLC3001 Cohort 1, which analyzed data from 266 patients with metastatic urothelial carcinoma who harbored selected FGFR3 alterations and who were treated with 1 or 2 prior systemic treatments including a PD-1 or PD-L1 inhibitor. In this open-label trial, patients were randomized 1:1 to receive either erdafitinib or investigator’s choice of chemotherapy, particularly vinflunine or docetaxel. Randomization was stratified by performance status, region, and presence of visceral or bone metastases, according to the FDA’s notice.

FGFR3 alterations were identified using the therascreen FGFR RGQ RT-PCR kit, which analyzes tumor tissue in a central laboratory, in 75% of patients, whereas the other patients underwent local next-generation sequencing assays.

The major efficacy outcome measure in this study was overall survival. Other outcome measures were included such as objective response rate and investigator-assessed progression-free survival.

Compared with chemotherapy, patients assigned erdafitinib obtained a statistically significant improvement in overall survival, progression-free survival and overall response rates. In particular, median overall survival was 12.1 months (95% CI, 10.3-16.4) in patients assigned erdafitinib compared with 7.8 months (95% CI, 6.5-11.1) in those assigned chemotherapy (HR = 0.64; 95% CI, 0.47-0.88; P = .0050). The erdafitinib group had a median progression-free survival of 5.6 months (95% CI, 4.4-5.7) compared with 2.7 months (95% CI, 1.8-3.7) in the chemotherapy group (HR = 0.58; 95% CI, 0.44-0.78; P = .0002). Patients receiving erdafitinib had a confirmed overall response rate of 35.3% (95% CI, 27.3%-43.9%) vs 8.5% (95% CI, 4.3%-14.6%) in those receiving chemotherapy (P < .001).

The most common adverse reactions, occurring in at least 20% of patients in the study, included nail disorders, increased phosphate, stomatitis, diarrhea, decreased hemoglobin, increased alkaline phosphatase, increased aspartate aminotransferase, increased alanine aminotransferase, increased creatinine, decreased sodium, decreased phosphate, dry mouth, dysgeusia, palmar-plantar erythrodysesthesia, fatigue, constipation, dry skin, increased calcium, decreased appetite, dry eye, alopecia, decreased weight, and increased potassium.

The FDA noted that the recommended dose of erdafitinib is 8 mg orally once per day, and increasing the dose to 9 mg once per day based on a patient’s tolerability, including hyperphosphatemia, at 14 to 21 days. Patients may continue treatment with erdafitinib until unacceptable toxicity or disease progression.


FDA approves erdafitinib for locally advanced or metastatic urothelial carcinoma. FDA. January 19, 2024. Accessed January 19, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-erdafitinib-locally-advanced-or-metastatic-urothelial-carcinoma

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