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Erdafitinib in Asian patients with advanced solid tumors: an open-label, single-arm, phase IIa trial
Joon Oh Park1, Yin-Hsun Feng2, Wu-Chou Su3
1Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center Sungkyunkwan University School of Medicine, Seoul, Korea. oncopark@skku.edu.
Background:
FGFR genomic aberrations occur in approximately 5-10% of human cancers. Erdafitinib has previously demonstrated efficacy and safety in FGFR-altered advanced solid tumors, such as gliomas, thoracic, gastrointestinal, gynecological, and other rare cancers. However, its efficacy and safety in Asian patients remain largely unknown. We conducted a multicenter, open-label, single-arm phase IIa study of erdafitinib to evaluate its efficacy in Asian patients with FGFR-altered advanced cholangiocarcinoma, non-small cell lung cancer (NSCLC), and esophageal cancer.
Methods:
Patients with pathologically/cytologically confirmed, advanced, or refractory tumors who met molecular and study eligibility criteria received oral erdafitinib 8 mg once daily with an option for pharmacodynamically guided up-titration to 9 mg on a 28-day cycle, except for four NSCLC patients who received erdafitinib 10 mg (7 days on/7 days off) as they were recruited before the protocol amendment. The primary endpoint was investigator-assessed objective response rate per RECIST v1.1. Secondary endpoints included progression-free survival, duration of response, disease control rate, overall survival, safety, and pharmacokinetics.
Results:
Thirty-five patients (cholangiocarcinoma: 22; NSCLC: 12; esophageal cancer: 1) were enrolled. At data cutoff (November 19, 2021), the objective response rate for patients with cholangiocarcinoma was 40.9% (95% CI, 20.7-63.6); the median progression-free survival was 5.6 months (95% CI, 3.6-12.7) and median overall survival was 40.2 months (95% CI, 12.4-not estimable). No patient with RET/FGFR-altered NSCLC achieved objective response and the disease control rate was 25.0% (95% CI, 5.5-57.2%), with three patients with stable disease. The single patient with esophageal cancer achieved partial response. All patients experienced treatment-emergent adverse events, and grade ≥ 3 treatment-emergent adverse events were reported in 22 (62.9%) patients. Hyperphosphatemia was the most frequently reported treatment-emergent adverse event (all-grade, 85.7%).
Conclusions:
Erdafitinib demonstrated efficacy in a population of Asian patients in selected advanced solid tumors, particularly in those with advanced FGFR-altered cholangiocarcinoma. Treatment was tolerable with no new safety signals.
Trial Registration:
This trial is registered with ClinicalTrials.gov (NCT02699606); study registration (first posted): 04/03/2016.
Insights
Erdafitinib showed efficacy in Asian patients with advanced FGFR-altered cholangiocarcinoma, non-small cell lung cancer, and esophageal cancer. The treatment was tolerable, with hyperphosphatemia being the most common side effect.
Area of Science:
- Oncology
- Pharmacology
- Genetics
Background:
- Fibroblast growth factor receptor (FGFR) genomic aberrations are present in 5-10% of human cancers.
- Erdafitinib has shown prior efficacy in FGFR-altered advanced solid tumors.
- Data on erdafitinib's efficacy and safety in Asian populations were limited.
Purpose of the Study:
- To evaluate the efficacy and safety of erdafitinib in Asian patients with advanced solid tumors harboring FGFR alterations.
- Specifically assessed in cholangiocarcinoma, non-small cell lung cancer (NSCLC), and esophageal cancer.
Main Methods:
- A multicenter, open-label, single-arm phase IIa study was conducted.
- Patients received oral erdafitinib with potential dose up-titration.
- Primary endpoint was investigator-assessed objective response rate (ORR) per RECIST v1.1.
Main Results:
- Twenty-two cholangiocarcinoma patients had an ORR of 40.9% (95% CI, 20.7-63.6) and median overall survival of 40.2 months.
- Twelve NSCLC patients showed no objective response, with a disease control rate of 25.0%.
- All patients experienced treatment-emergent adverse events, most commonly hyperphosphatemia (85.7%).
Conclusions:
- Erdafitinib demonstrated efficacy in Asian patients with advanced FGFR-altered cholangiocarcinoma.
- The drug was tolerable, with no new safety concerns identified.
- Further investigation in specific cancer types like cholangiocarcinoma is warranted.
