Recordati B V — Qarziba (previously Dinutuximab beta EUSA and Dinutuximab beta Apeiron) EMA EPAR public assessment report (EMEA/H/C/003918, 2017)

Report summary

This EMA EPAR report details the regulatory approval of Dinutuximab beta (Qarziba) for high-risk neuroblastoma. The drug, an orphan medicinal product, underwent a centralized procedure, with its marketing authorization simplified by the withdrawal of Unituxin. The report covers the product's mechanism of action, manufacturing process, and extensive quality control, including comparability studies and stability data. Non-clinical and clinical studies demonstrate its efficacy, particularly in recurrent and high-risk forms, showing improved survival rates compared to historical controls. Pharmacokinetic and pharmacodynamic data, though sometimes limited by assay issues, confirm its GD2 binding and immune-mediated anti-tumor activity. The safety profile indicates a high incidence of adverse events, including pain and hematological abnormalities. Despite manufacturing differences from Unituxin, it was not deemed a new active substance due to insufficient evidence of significant safety/efficacy differences.

Key findings — cited to the page