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Derazantinib Showed Clinical Benefit as a Second-Line Treatment for Patients With Intrahepatic Cholangiocarcinoma and EGFR2 Aberrations
In a phase 2 study, derazantinib, a potent FGFR1-3 kinase inhibitor, demonstrated meaningful clinical benefit for patients with intrahepatic cholangiocarcinoma and FGFR2 genetic aberrations, including FGFR2F and FGFR2MA. For this patient population, there are “limited options after progression after [first-line] chemotherapy,” Mitesh Borad, MD, Mayo Clinic Cancer Center, Scottsdale, Arizona, and colleagues stated.
The open-label, single-arm phase 2 FIDES-01 study, enrolled 143 patients with inoperable or advanced intrahepatic cholangiocarcinoma and FGFR2 gene fusions, or FGFR2 gene mutations or amplifications, who had previously received at least 1 regimen of systemic therapy. All patients received 300 mg derazantinib once daily.
The primary endpoint was objective response rate (ORR) for patients with FGFR2F and progression-free survival (PFS) for patients with FGFR2MA. Response and safety was evaluable in 103 patients with FGFR2. For patients with FGFR2MA, safety was evaluable in 40 patients and response was evaluable in 31.
At the data cutoff date of March 25, 2022, the ORR of patients with FGFR2F was 21.4% (95% confidence interval [CI], 0.8 to 21.4) and the median PFS was 8.0 months (95% CI, 5.5 to 8.3). The ORR of patients with FGFR2MA was 6.5% (95% CI, 0.8 to 21.4) and the median PFS was 8.3 months (95% CI, 1.9 to 16.7).
The most common grade ≥3 adverse drug reactions were transaminase elevations (12%), asthenia/fatigue (5%), and hyperphosphatemia (3%). Instances of drug-related nail toxicities, retinal events, stomatitis, and palmar-plantar erythrodysesthia were rare.
Source:
Borad M, Javle M, Shaib WL, et al. 59P Efficacy of derazantinib in intrahepatic cholangiocarcinoma (iCCA) patients with FGFR2 fusions, mutations or amplifications. Annals Oncol. 2022;33(7):S567-S568. doi:10.1016/j.annonc.2022.07.087
