GSK's investigational cancer drug receives Orphan Drug Designation in Japan
GSK plc announced on March 23, 2026, that risvutatug rezetecan has received Orphan Drug Designation from Japan's Ministry of Health, Labour and Welfare for small-cell lung cancer. Early clinical data supported the designation.
GSK plc announced on March 23, 2026, that its B7-H3-targeted antibody-drug conjugate, risvutatug rezetecan, has received Orphan Drug Designation from Japan's Ministry of Health, Labour and Welfare for the treatment of small-cell lung cancer (SCLC).
The designation was supported by preliminary clinical data demonstrating durable responses in patients with extensive-stage SCLC treated with risvutatug rezetecan in the Phase I ARTEMIS-001 clinical trial. Extensive-stage SCLC is associated with high relapse rates and limited treatment options.
Risvutatug rezetecan is an investigational antibody-drug conjugate developed by GSK, acquired from Hansoh Pharma. This marks the sixth global regulatory designation for the drug, which is being developed for various solid tumors.
GSK initiated a global Phase III trial for risvutatug rezetecan in relapsed extensive-stage SCLC in August 2025. The company is seeking to advance the development of treatments for difficult-to-treat cancers.