On December 10, 2025 GSK plc (LSE/NYSE: GSK) reported that its B7-H3-targeted antibody-drug conjugate GSK’227, now referred to by its International Nonproprietary Name, risvutatug rezetecan, has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for the treatment of 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 risvutatug rezetecan in the phase I ARTEMIS-001 clinical trial.1
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In the US, SCLC constitutes about 13% of all lung cancers. In 2025, an estimated 29,500 people in the US will be diagnosed with SCLC.2 Of patients with SCLC, 70% have extensive-stage disease, meaning the cancer has spread throughout one or both lungs and/or to other parts of the body.3 ES-SCLC is an aggressive and difficult-to-treat cancer with limited treatment options. The 5-year survival rate is approximately 3%.3 Most patients with ES-SCLC relapse after initial treatment and the median overall survival with standard-of-care treatments for relapsed ES-SCLC is approximately 8 months.4
This designation follows the recent announcement that risvutatug rezetecan was granted ODD from the European Medicines Agency (EMA) for the treatment of pulmonary neuroendocrine carcinoma, a category of cancer that includes SCLC. It is the fifth regulatory designation for risvutatug rezetecan, exemplifying the potential of this B7-H3-targeted ADC, which is being developed in a range of solid tumours, including lung, prostate and colorectal cancers. Previously, risvutatug rezetecan 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.5,6,7
About risvutatug rezetecan
Risvutatug rezetecan (GSK5764227) 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 risvutatug rezetecan. GSK’s global phase III trial (NCT07099898) for risvutatug rezetecan in relapsed ES-SCLC began in August 2025.
(Press release, GlaxoSmithKline, DEC 10, 2025, View Source [SID1234661344])