FDA Approves Erdafitinib for Patients With Locally Advanced/Metastatic Urothelial Carcinoma Harboring FGFR3 Alterations

On January 19, 2024, the US Food and Drug Administration (FDA) approved erdafitinib for patients with locally advanced/metastatic urothelial carcinoma with susceptible FGFR3 genetic alterations as determined by an FDA-approved companion diagnostic test. This regulatory decision was based on results from cohort 1 of the BLC3001 study.

In this open-label study, 266 patients with metastatic urothelial carcinoma harboring selected FGFR3 alterations who previously received 1 to 2 lines of systemic therapy with a PD-1/PD-L1 inhibitor were randomized on a 1-to-1 basis to receive either 8 mg of erdafitinib or investigator’s choice of docetaxel or vinflunine daily. Randomization was stratified based on region, performance status, and presence of visceral or bone metastases. FGFR3 alterations were identified using the therascreen FGFR RGQ RT-PCR kit in 75% of patients, and local next-generation sequencing assays in the remaining patients. The major efficacy outcome measure was overall survival (OS). Secondary efficacy measures included progression-free survival (PFS) and objective response rate (ORR).

At analysis, median OS was 12.1 months in the erdafitinib arm and 7.8 months in the chemotherapy arm (hazard ratio [HR], 0.64; 95% confidence interval [CI], 0.47 to 0.88; P = .0050). Median PFS was 5.6 months in the erdafitinib arm and 2.7 months in the chemotherapy arm (HR 0.58; 95% CI, 0.44 to 0.78; P = .0002). Confirmed ORR was 35.3% in the erdafitinib arm and 8.5% in the chemotherapy arm (P < .001).

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

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

This approval amends the accelerated approval previously granted for patients with metastatic urothelial carcinoma with susceptible FGFR3/FGFR2 alterations after prior platinum-containing chemotherapy.

Source:

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