Balversa(erdafitinib)

Balversa (erdafitinib) is indicated 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. It was qualified as a new active substance and received a CHMP positive opinion for marketing authorisation on 27 June 2024, following a Marketing Authorisation Application submission on 8 September 2023.

In a Phase 3 study (BLC3001), Balversa demonstrated a median Progression-Free Survival of 5.55 months compared to 2.73 months for chemotherapy (vinflunine or docetaxel), with a Progression-Free Survival Hazard Ratio of 0.58 (P-value 0.0002). The Objective Response Rate (CR + PR) for Balversa was 45.6% compared to 11.5% for chemotherapy, with an Objective Response Rate Relative Risk of 3.94 (P-value <.001). Median Overall Survival was 12.06 months for Balversa versus 7.79 months for chemotherapy, with an Overall Survival Hazard Ratio of 0.64 (P-value 0.0050).

Key safety findings include Grade 3 or 4 TEAEs in 64% of patients, Serious AEs in 40.6%, and TEAEs leading to discontinuation in 16.9%. Notable adverse events included hyperphosphataemia (78.6%), gastrointestinal disorders (82.8%), nail disorders (62.3%), and central serous retinopathy (20.1%).