GSK’s experimental lung cancer drug gains FDA orphan drug status

GSK holds exclusive global rights (excluding mainland China, Hong Kong, Macau, and Taiwan) from Hansoh Pharma to advance its development and commercialization

GSK has announced that its B7-H3-targeted antibody-drug conjugate, GSK’227—now officially known as risvutatug rezetecan—has received Orphan Drug Designation (ODD) from the US FDA for the treatment of small-cell lung cancer (SCLC). 

The designation is backed by early clinical data showing durable responses in patients with extensive-stage SCLC (ES-SCLC) treated in the phase I ARTEMIS-001 trial. 

In the United States, SCLC accounts for about 13% of all lung cancers, with an estimated 29,500 diagnoses expected in 2025. Of these, 70% have extensive-stage disease, meaning the cancer has spread throughout one or both lungs or to other parts of the body. 

S-SCLC is highly aggressive with limited treatment options, a 5-year survival rate of roughly 3%, and a median overall survival of approximately 8 months with standard-of-care treatments, the global pharma giant said. 

This FDA designation follows recent EMA Orphan Drug recognition for risvutatug rezetecan in pulmonary neuroendocrine carcinoma, which includes SCLC. It marks the fifth regulatory nod for the investigational therapy, highlighting its potential across multiple solid tumors, including lung, prostate, and colorectal cancers. 

Previously, risvutatug rezetecan earned Priority Medicines (PRIME) Designation from the EMA for relapsed or refractory ES-SCLC, as well as Breakthrough Therapy Designations from the FDA for relapsed or refractory ES-SCLC and relapsed or refractory osteosarcoma. 

Risvutatug rezetecan is a novel B7-H3-targeted antibody-drug conjugate, made of a fully human anti-B7-H3 monoclonal antibody linked to a topoisomerase inhibitor payload. GSK holds exclusive global rights (excluding mainland China, Hong Kong, Macau, and Taiwan) from Hansoh Pharma to advance its development and commercialization.