GSK’s B7-H3-targeted ADC, Risvutatug rezetecan, granted Orphan Drug Designation for small-cell lung cancer in Japan

CAR-T Digest - August 2020

23 March 2026: GSK’s B7-H3-targeted ADC, Risvutatug rezetecan, granted Orphan Drug Designation for small-cell lung cancer in Japan

GSK’s investigational B7-H3-targeted ADC, risvutatug rezetecan (Ris-Rez), has received ODD in Japan for the treatment of small-cell lung cancer, further expanding its global regulatory momentum
The decision is supported by early clinical data from the Phase 1 ARTEMIS-001 trial, where risvutatug rezetecan demonstrated durable responses in patients with extensive-stage SCLC, indicating promising antitumor activity in a heavily pretreated population
This designation addresses a significant unmet need, as extensive-stage SCLC remains an aggressive disease with high relapse rates, limited treatment options, and a median overall survival of approximately eight months under current standard of care
The asset is a B7-H3-directed antibody-drug conjugate with a topoisomerase inhibitor payload, and is currently being advanced into a global Phase 3 trial in relapsed extensive-stage SCLC, reflecting confidence in its clinical potential
With this latest designation, risvutatug rezetecan has now received multiple global regulatory recognitions, reinforcing its positioning as a next-generation ADC with potential across multiple solid tumors, including lung, prostate, and colorectal cancers