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Erdafitinib in Patients with FGFR-Altered Advanced or Metastatic Cholangiocarcinoma
Shubham Pant1, Joon Oh Park2, Wu-Chou Su3
1The University of Texas MD Anderson Cancer Center , Houston, Texas.
Purpose:
Up to 20% of patients with cholangiocarcinoma (CCA) harbor FGFR gene aberrations. Erdafitinib is approved for pretreated, locally advanced/metastatic urothelial carcinoma with susceptible FGFR3 alterations. The present study evaluated the efficacy and safety of erdafitinib using a pooled analysis of patients with CCA from the RAGNAR and LUC2001 studies.
Experimental Design:
In RAGNAR (phase II, global, tumor-agnostic study) and LUC2001 (an open-label, multicenter, phase IIa study in Asian patients), patients with advanced solid tumors after ≥1 prior lines of therapy received once daily oral erdafitinib (8 mg/day with an option for pharmacodynamically guided up-titration to 9 mg). Patients were pooled for efficacy [objective response rate (ORR) per blinded independent review committee, duration of response (DOR), progression-free survival (PFS), and overall survival (OS)] and safety analyses.
Results:
At a median efficacy follow-up of 14.7 months in 78 erdafitinib-treated patients (RAGNAR: n = 66; LUC2001: n = 12), ORR was 55% [95% confidence interval (CI), 43.4-66.4]. The median time to response was 1.7 months; the median DOR, PFS, and OS were 6.9 (95% CI, 4.37-8.61), 8.5 (95% CI, 6.83-9.72), and 18.1 (95% CI, 13.40-24.28) months, respectively. The most common treatment-emergent adverse events (TEAE) were hyperphosphatemia (83%), stomatitis (72%), diarrhea (68%), dry mouth (51%), and palmar-plantar erythrodysesthesia (51%); 42% had serious TEAEs, and 12% had TEAEs leading to treatment discontinuation.
Conclusions:
Pooled analyses confirm the robust efficacy of erdafitinib in a diverse population of pretreated patients with advanced/metastatic CCA harboring prespecified FGFR alterations. These findings are consistent with previously observed efficacy of FGFR-targeted agents in patients with CCA.
Insights
Erdafitinib demonstrated robust efficacy in patients with advanced cholangiocarcinoma (CCA) harboring FGFR alterations. This targeted therapy showed significant response rates and survival benefits in a pooled analysis of clinical trials.
Area of Science:
- Oncology
- Pharmacology
- Genetics
Background:
- Cholangiocarcinoma (CCA) presents a significant unmet need, with up to 20% of patients exhibiting FGFR gene aberrations.
- Erdafitinib is an approved FGFR inhibitor for urothelial carcinoma, suggesting potential utility in other FGFR-altered malignancies.
Purpose of the Study:
- To evaluate the efficacy and safety of erdafitinib in patients with advanced/metastatic cholangiocarcinoma (CCA) harboring FGFR alterations.
- To conduct a pooled analysis of data from the RAGNAR and LUC2001 studies to assess erdafitinib's therapeutic potential in CCA.
Main Methods:
- A pooled analysis of patients with advanced solid tumors, specifically CCA with FGFR alterations, from the RAGNAR and LUC2001 phase 2 studies.
- Patients received oral erdafitinib (8 mg/day, with optional up-titration to 9 mg) after at least one prior line of therapy.
- Efficacy endpoints included objective response rate (ORR), duration of response (DOR), progression-free survival (PFS), and overall survival (OS); safety was assessed via treatment-emergent adverse events (TEAEs).
Main Results:
- The pooled analysis included 78 patients treated with erdafitinib, with a median follow-up of 14.7 months.
- An objective response rate (ORR) of 55% was observed (95% CI: 43.4-66.4).
- Median DOR, PFS, and OS were 6.9, 8.5, and 18.1 months, respectively. Common TEAEs included hyperphosphatemia, stomatitis, and diarrhea; 12% discontinued treatment due to TEAEs.
Conclusions:
- Pooled analyses confirm the robust efficacy of erdafitinib in pretreated patients with advanced/metastatic CCA harboring FGFR alterations.
- These findings support the use of erdafitinib as a targeted therapy option for this patient population.
- The results align with the known efficacy of FGFR-targeted agents in CCA treatment.
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