Erdafitinib in Asian Patients with Advanced CCA and FGFR Alterations: Updated Analysis

March 2022, Vol 3, No 1

The ongoing open-label, multicenter, phase 2a LUC2001 study is investigating the efficacy and safety of the FGFR inhibitor erdafitinib in a molecularly defined subset of Asian patients with advanced cholangiocarcinoma (CCA) harboring FGFR alterations whose disease progressed after ≥1 previous systemic therapies.

Yin-Hsun Feng, MD, Division of Hematology and Oncology, Chi Mei Medical Center, Tainan, Taiwan, reported updated data at the 2022 ASCO GI Cancers Symposium.

Key inclusion criteria included age ≥18 years, histologically or cytologically confirmed diagnosis of advanced CCA and ≥1 FGFR gene fusions or mutations, ECOG performance status 0-1, and disease progression after ≥1 previous lines of systemic therapy. Eligible patients received erdafitinib 8 mg once daily, with pharmacodynamically guided up-titration to 9 mg once daily. The primary end point was objective response rate (ORR); the secondary end points included best overall response, disease control rate, duration of response (DOR), progression-free survival (PFS), overall survival (OS), and safety.

Of the 232 patients who had molecular screening, 39 (16.8%) patients had FGFR alterations, including 21 (9.1%) fusions and 19 (8.2%) mutations. Overall, 22 patients were evaluable in this analysis; the median age of this subset was 52 years (range, 29-69 years), the majority (59.1%) were male, 72.7% had malignant intrahepatic CCA, 22.7% had extrahepatic CCA, and 54.5% had received ≥2 previous lines of systemic therapy.

At a median follow-up of 22.4 months (median treatment duration, 6.2 months), the investigator-assessed ORR was 40.9% (N = 9), including 1 complete response and 8 partial responses, with a median time to response of 1.8 months (range, 1.5-5.6 months). The median DOR was 7.3 months (95% confidence interval [CI], 3.7-17.5), the median PFS was 5.6 months (95% CI, 3.6-12.7), and the median OS was 40.2 months (95% CI, 9.9-not estimable). Overall, 8 of 14 patients with FGFR fusions and 1 of 8 patients with FGFR mutations had a response to treatment with erdafitinib.

All 22 patients had ≥1 treatment-emergent adverse events (TEAEs); the most common TEAEs included dry mouth (68.2%), stomatitis (63.6%), increased alanine aminotransferase (ALT; 50%), aspartate aminotransferase (45.5%), and dry skin (45.4%). Grade ≥3 adverse events occurred in 68.2% of patients, of which 50% were TEAEs. The most common ≥3 TEAEs were stomatitis (13.6%) and increased ALT (13.6%). Serious TEAEs were reported in 50% of patients. An event of special interest was detachment of retinal pigment epithelium in 1 patient, and 1 patient died of sepsis (which was not treatment-related).

Based on these results, the researchers concluded that erdafitinib therapy was associated with durable efficacy and a manageable safety profile in patients with advanced CCA harboring FGFR alterations.


Feng YH, Su WC, Oh DY, et al. Updated analysis with longer follow up of a phase 2a study evaluating erdafitinib in Asian patients (pts) with advanced cholangiocarcinoma (CCA) and fibroblast growth factor receptor (FGFR) alterations. Abstract 430.

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