GSK’s B7-H3-targeted antibody-drug conjugate, GSK’227, receives Orphan Drug Designation in the EU

On October 28, 2025 GSK plc (LSE/NYSE: GSK) reported that GSK5764227 (GSK’227), its B7-H3-targeted antibody-drug conjugate (ADC), has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) for the treatment of pulmonary neuroendocrine carcinoma (NEC), a category of cancer that includes small-cell lung cancer (SCLC). The ODD was supported by preliminary clinical data showing durable responses in patients with extensive stage SCLC (ES-SCLC) who were treated with GSK’227 in the phase I ARTEMIS-001 clinical trial.1

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This ODD recognises the potential of GSK’227 to address a significant unmet need for ES-SCLC, an aggressive type of NEC with poor outcomes and limited treatment options. An estimated 250,000 patients globally are diagnosed with SCLC each year and it is responsible for approximately 200,000 deaths annually.2

This is the fourth regulatory designation for GSK’227, exemplifying the potential of this targeted ADC, which is being developed in a range of solid tumour types, including in lung, colorectal, head and neck, and prostate cancers. Previously, GSK’227 was granted Priority Medicines (PRIME) designation by the EMA for relapsed or refractory ES-SCLC and Breakthrough Therapy Designations for relapsed or refractory ES-SCLC and relapsed or refractory osteosarcoma granted by the US FDA.3,4,5

About GSK’227
GSK’227 is a novel investigational B7-H3-targeted antibody-drug conjugate composed of a fully human anti-B7-H3 monoclonal antibody covalently linked to a topoisomerase inhibitor payload. GSK acquired exclusive worldwide rights (excluding China’s mainland, Hong Kong, Macau, and Taiwan) from Hansoh Pharma to progress clinical development and commercialisation of GSK’227. GSK’s global phase III trial for GSK’227 in relapsed ES-SCLC began in August 2025.

(Press release, GlaxoSmithKline, OCT 28, 2025, View Source [SID1234657067])