GSK's B7-H3-Targeted Antibody-Drug Conjugate Receives Orphan Drug Designation in the EU

GSK plc announced that its B7-H3-targeted antibody-drug conjugate, GSK'227, has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) for the treatment of pulmonary neuroendocrine carcinoma, including small-cell lung cancer (SCLC). This designation is based on encouraging early clinical data from the phase I ARTEMIS-001 trial, which showed durable responses in patients with extensive stage SCLC (ES-SCLC). ES-SCLC is an aggressive cancer with poor outcomes and limited treatment options, affecting approximately 250,000 patients globally each year. This is the fourth regulatory designation for GSK'227, which has also received Priority Medicines (PRIME) designation from the EMA and Breakthrough Therapy Designations from the US FDA for relapsed or refractory ES-SCLC and osteosarcoma. GSK'227 is currently in a global phase III trial for relapsed ES-SCLC, which began in August 2025.