Business
GSK Lung Cancer ADC Gets Japan Orphan Drug Status
GSK plc announced that its B7-H3-targeted antibody-drug conjugate, risvutatug rezetecan, has received Orphan Drug Designation in Japan for the treatment of small-cell lung cancer (SCLC), supported by early clinical data showing durable responses in extensive-stage SCLC patients. This marks the sixth global regulatory designation for risvutatug rezetecan, which is being developed for various solid tumours and has previously received designations from the US FDA and EMA, including Breakthrough Therapy and PRIME designations for SCLC. Disclaimer*
About this update from Gsk Plc
[{"type":"text","content":"\n\nIssued: 23 March 2026, London UK\n \nGSK's B7-H3-targeted antibody-drug conjugate, risvutatug rezetecan, granted Orphan Drug Designation for small-cell lung cancer in Japan\n \n· Designation supported by early clinical data showing durable responses in certain types of small-cell lung cancer (SCLC)\n\n· Extensive-stage SCLC is associated with high rates of relapse, few treatment options and poor prognosis\n\n· Sixth global regulatory designation for risvutatug rezetecan supporting GSK's investigational ADC with transformational potential in solid tumours\n \nGSK plc (LSE/NYSE: GSK) today announced that risvutatug rezetecan (or Ris-Rez, for short), a B7-H3-targeted antibody-drug conjugate (ADC), has received Orphan Drug Designation (ODD) from Japan's Ministry of Health, Labour and Welfare 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 Ris-Rez in the phase I ARTEMIS-001 clinical trial.[1] This is the sixth regulatory designation for Ris-Rez, which is being developed in a range of solid tumours, including lung, prostate and colorectal cancers.\n \nLung cancer is the second most common cancer in Japan, and SCLC makes up 10-15% of cases.[2],[3] Of patients with SCLC, 70% have ES-SCLC, meaning the cancer has spread throughout one or both lungs and/or to other parts of the body.[4] ES-SCLC is an aggressive and difficult-to-treat cancer with limited treatment options and poor long-term survival. Most patients with ES-SCLC relapse after initial treatment and the median overall survival with standard-of-care for these patients is approximately 8 months.[5]\n \nAbout Ris-Rez\nRis-Rez 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 Ris-Rez. GSK's global phase III trial (NCT07099898) for Ris-Rez in relapsed ES-SCLC began in August 2025.\n \nRegulatory designations received for Ris-Rez to date include ODDs from the US Food ...