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FDA Approval Summary: Erdafitinib for FGFR3-Altered Locally Advanced or Metastatic Urothelial Carcinoma
William F Maguire1, Elaine Chang1, Flora Mulkey1
1United States Food and Drug Administration Silver Spring, MD United States.
Abstract:
On January 19, 2024, the FDA granted traditional approval to erdafitinib for patients with locally advanced or metastatic urothelial carcinoma (la/mUC) with susceptible FGFR3 genetic alterations, as determined by an FDA-approved companion diagnostic test, whose disease has progressed on or after at least one line of prior systemic therapy. Substantial evidence of effectiveness was obtained from BLC3001 (THOR, NCT03390504) Cohort 1, which was an open-label trial in which 266 patients with FGFR3-altered la/mUC were randomized 1:1 to receive erdafitinib (8 mg once daily with potential up-titration to 9 mg) versus chemotherapy (docetaxel 75 mg/m2 every 3 weeks or vinflunine 320 mg/m2 every 3 weeks) until disease progression or unacceptable toxicity. The primary endpoint was overall survival (OS). Statistically significant improvements in OS, progression-free survival, and objective response rate were demonstrated for erdafitinib compared with chemotherapy. Median OS was 12.1 months (95% CI: 10.3, 16.4) in the erdafitinib arm and 7.8 months (95% CI: 6.5, 11.1) in the chemotherapy arm (HR: 0.64, 95% CI: 0.47, 0.88; p=0.0050). Based on BLC3001 Cohort 2 results showing no OS benefit of erdafitinib versus pembrolizumab (HR 1.18, 95% CI: 0.92, 1.51), the approval includes a limitation of use: erdafitinib is not recommended for the treatment of patients who are eligible for and have not received prior PD-(L)1 inhibitor therapy. This article summarizes the data and the FDA thought process supporting traditional approval of erdafitinib, including the rationale for not requiring specific types of prior systemic therapy in the indication statement.
Insights
Erdafitinib is now FDA-approved for advanced urothelial carcinoma with FGFR3 alterations. This targeted therapy demonstrated improved overall survival compared to chemotherapy in clinical trials.
Area of Science:
- Oncology
- Genetics
- Pharmacology
Background:
- Locally advanced or metastatic urothelial carcinoma (la/mUC) with susceptible FGFR3 genetic alterations presents a treatment challenge.
- Prior systemic therapies have limited efficacy in this patient population.
- Targeted therapies offer a promising approach for specific genetic alterations.
Purpose of the Study:
- To summarize the data supporting the traditional FDA approval of erdafitinib for la/mUC.
- To evaluate the efficacy and safety of erdafitinib compared to chemotherapy in patients with FGFR3-altered la/mUC.
- To outline the FDA's rationale for the approved indication and limitations of use.
Main Methods:
- A randomized, open-label trial (BLC3001 Cohort 1) comparing erdafitinib to chemotherapy (docetaxel or vinflunine) in 266 patients with FGFR3-altered la/mUC.
- Patients received treatment until disease progression or unacceptable toxicity.
- Primary endpoint was overall survival (OS).
Main Results:
- Erdafitinib demonstrated statistically significant improvements in OS (median 12.1 vs. 7.8 months; HR: 0.64), progression-free survival, and objective response rate compared to chemotherapy.
- However, erdafitinib showed no OS benefit versus pembrolizumab in a separate cohort (BLC3001 Cohort 2).
- The approval includes a limitation of use, excluding patients eligible for prior PD-(L)1 inhibitor therapy.
Conclusions:
- Erdafitinib has received traditional FDA approval for patients with advanced urothelial carcinoma harboring FGFR3 alterations who have progressed on prior systemic therapy.
- Erdafitinib offers a survival benefit over chemotherapy for this specific patient group.
- The indication is limited, not recommending erdafitinib for patients who have not received prior PD-(L)1 inhibitor therapy.
