GSK’s risvutatug rezetecan granted orphan drug designation for SCLC in Japan
- Risvutatug rezetecan is a B7-H3-targeted antibody-drug conjugate with a topoisomerase inhibitor payload.
- GSK holds exclusive global rights (excluding mainland China, Hong Kong, Macau and Taiwan) acquired from Hansoh Pharma.
- Global phase III trial NCT07099898 in relapsed extensive-stage SCLC began in August 2025.
- Japan's Ministry of Health, Labour and Welfare granted Orphan Drug Designation for SCLC; Ris-Rez previously received US FDA ODD and Breakthrough Therapy designations and EMA PRIME.
Designation and supporting data
Japan’s Ministry of Health, Labour and Welfare granted Orphan Drug Designation to risvutatug rezetecan for small‑cell lung cancer, based on preliminary phase I ARTEMIS‑001 data showing durable responses in extensive‑stage SCLC patients.
Disease context
SCLC accounts for about 10–15% of lung cancers in Japan and roughly 70% of cases are extensive stage, which has high relapse rates, limited treatment options and a median overall survival of approximately 8 months with standard care.
Ris‑Rez mechanism and rights
Risvutatug rezetecan is a B7‑H3‑targeted antibody‑drug conjugate composed of a fully human anti‑B7‑H3 monoclonal antibody covalently linked to a topoisomerase inhibitor payload; GSK holds exclusive worldwide rights excluding mainland China, Hong Kong, Macau and Taiwan, acquired from Hansoh Pharma.
Regulatory status and clinical development
A global phase III trial (NCT07099898) in relapsed extensive‑stage SCLC began in August 2025; prior regulatory recognitions include US FDA and EMA orphan drug designations, EMA PRIME for relapsed/refractory ES‑SCLC, and US FDA Breakthrough Therapy designations for relapsed/refractory ES‑SCLC and late‑line osteosarcoma.
Source: GSK