Regulatory approval

Published by the Health Service Executive.

The Republic of Ireland's Health Service Executive (HSE) has approved erdafitinib as a treatment option for the treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), harbouring susceptible FGFR3 genetic alterations who have previously received at least one line of therapy containing a PD-1 or PD-L1 inhibitor in the unresectable or metastatic treatment setting. This indication is based on BLC3001 Study Cohort 1, a Phase 3, randomised, open-label, multicentre study, where eligible patients were required to have at least one of the following FGFR fusions: FGFR2::BICC1, FGFR2::CASP7, FGFR3::TACC3, FGFR3::BAIAP2L1; or 1 of the following FGFR3 gene mutations: R248C, S249C, G370C, Y373C.

This is written in the approval document as:

As monotherapy for the treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), harbouring susceptible FGFR3 genetic alterations who have previously received at least one line of therapy containing a PD-1 or PD-L1 inhibitor in the unresectable or metastatic treatment setting.

Citation

Erdafitinib Monotherapy, 2024, version number 1a, viewed 01/10/2025, https://www.hse.ie/eng/services/list/5/cancer/profinfo/chemoprotocols/oral-anti-cancer-medicines/885-erdafitinib-monotherapy.pdf

Therapeutic response

Precision oncology relationships for therapeutic response derived from this regulatory approval.

Type Biomarker(s) Cancer type Therapy(ies)
Sensitivity (+) FGFR2::BICC1 Bladder Urothelial Carcinoma Erdafitinib
Sensitivity (+) FGFR2::CASP7 Bladder Urothelial Carcinoma Erdafitinib
Sensitivity (+) FGFR3::TACC3 Bladder Urothelial Carcinoma Erdafitinib
Sensitivity (+) FGFR3::BAIAP2L1 Bladder Urothelial Carcinoma Erdafitinib
Sensitivity (+) FGFR3 p.R248C Bladder Urothelial Carcinoma Erdafitinib
Sensitivity (+) FGFR3 p.S249C Bladder Urothelial Carcinoma Erdafitinib
Sensitivity (+) FGFR3 p.G370C Bladder Urothelial Carcinoma Erdafitinib
Sensitivity (+) FGFR3 p.Y373C Bladder Urothelial Carcinoma Erdafitinib