FDA Approval Summary: Erdafitinib for FGFR3-Altered Locally Advanced or Metastatic Urothelial Carcinoma.

Food and Drug Administration (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.

从BLC3001（THOR，NCT03390504）队列1中获得了显著的有效性证据，这是一项开放标签试验，其中266名具有FGFR3改变的la/mUC患者被随机1:1分配接受Erdafitinib（每日一次8毫克，可能增加至9毫克）或化疗（每3周一次的多西他赛75毫克/平方米或每3周一次的vinflunine 320毫克/平方米），直到疾病进展或出现不可接受的毒性。

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 uptitration to 9 mg) compared with chemotherapy (docetaxel 75 mg/m2 every 3 weeks or vinflunine 320 mg/m2 every 3 weeks) until disease progression or unacceptable toxicity.

从BLC3001（THOR，NCT03390504）队列1中获得了显著的有效性证据，这是一项开放标签试验，其中266名具有FGFR3改变的la/mUC患者被随机1:1分配接受Erdafitinib（每日一次8毫克，可能增加至9毫克）或化疗（每3周一次的多西他赛75毫克/平方米或每3周一次的vinflunine 320毫克/平方米），直到疾病进展或出现不可接受的毒性。

The primary endpoint was overall survival(OS).

主要终点是总生存期（OS）。

Statistically significant improvements in OS, progression-free survival, and objective response ratewere demonstrated for erdafitinib compared with chemotherapy.

与化疗相比，Erdafitinib在总生存期、无进展生存期和客观缓解率方面显示出统计学上的显著改善。

The median OS was 12.1 months [95% confidence interval(CI), 10.3-16.4] in the erdafitinib arm and 7.8 months (95% CI, 6.5-11.1) in the chemotherapy arm [hazard ratio(HR), 0.64; 95% CI, 0.47-0.88; P = 0.0050].

Erdafitinib组的中位总生存期为12.1个月[95%置信区间（CI），10.3-16.4]，化疗组为7.8个月（95% CI，6.5-11.1）[风险比（HR），0.64；95% CI，0.47-0.88；P = 0.0050]。

Based on BLC3001 Cohort 2 results showing no OS benefit of erdafitinib compared with 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.

基于BLC3001队列2的结果显示，Erdafitinib与pembrolizumab相比没有总生存期获益（HR，1.18；95% CI，0.92-1.51），批准中包含了一项使用限制：不推荐Erdafitinib用于适合且未接受过PD-(L)1抑制剂治疗的患者。

This article summarizes the data and the FDA thought process supporting the traditional approval of erdafitinib, including the rationale for not requiring specific types of prior systemic therapy in the indication statement.

本文总结了支持Erdafitinib传统批准的数据和FDA的思考过程，包括在适应症声明中不要求特定类型的先前系统治疗的理由。