GSK’227, a B7-H3-targeted antibody-drug conjugate, granted Orphan Drug Designation in small-cell lung cancer by the US FDA

CAR-T Digest - August 2020

10 December 2025: GSK’227, a B7-H3-targeted antibody-drug conjugate, granted Orphan Drug Designation in small-cell lung cancer by the US FDA

GSK’s B7-H3–targeted antibody-drug conjugate, Risvutatug rezetecan (GSK’227), has gained fresh regulatory momentum, receiving Orphan Drug Designation from the US FDA for the treatment of small-cell lung cancer (SCLC)
The designation is backed by encouraging early clinical signals, as preliminary data from the Phase 1 ARTEMIS-001 trial showed durable responses in patients with extensive-stage SCLC, an aggressive form of disease with few effective treatment options
This recognition comes at a critical time, with SCLC representing 13% of US lung cancer cases and up to 70% of patients presenting with extensive-stage disease, where relapse is common and five-year survival remains just 3%
The new FDA status follows a series of recent global acknowledgments, including EMA’s ODD for pulmonary neuroendocrine carcinoma, marking the fifth major regulatory designation awarded to risvutatug rezetecan
Together, these designations reinforce the therapeutic promise of this B7-H3–targeted ADC, which is being developed across multiple solid tumors including lung, prostate, and colorectal cancers, and has already earned PRIME and Breakthrough Therapy status for relapsed or refractory ES-SCLC and osteosarcoma