GSK risvutatug rezetecan gets USFDA Orphan Drug Designation for lung cancer

Written By :  Ruchika Sharma
Medically Reviewed By :  Dr. Kamal Kant Kohli
Published On 2025-12-11 05:00 GMT   |   Update On 2025-12-11 05:01 GMT
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London: GSK plc has announced that its B7-H3-targeted antibody-drug conjugate GSK'227, now referred to by its International Nonproprietary Name, risvutatug rezetecan, has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for the treatment of small-cell lung cancer (SCLC).

The ODD was supported by preliminary clinical data showing durable responses in patients with extensive stage SCLC (ES-SCLC) who were treated with risvutatug rezetecan in the phase I ARTEMIS-001 clinical trial.

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In the US, SCLC constitutes about 13% of all lung cancers. In 2025, an estimated 29,500 people in the US will be diagnosed with SCLC. Of patients with SCLC, 70% have extensive-stage disease, meaning the cancer has spread throughout one or both lungs and/or to other parts of the body. ES-SCLC is an aggressive and difficult-to-treat cancer with limited treatment options. The 5-year survival rate is approximately 3%. Most patients with ES-SCLC relapse after initial treatment and the median overall survival with standard-of-care treatments for relapsed ES-SCLC is approximately 8 months.

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This designation follows the recent announcement that risvutatug rezetecan was granted ODD from the European Medicines Agency (EMA) for the treatment of pulmonary neuroendocrine carcinoma, a category of cancer that includes SCLC. It is the fifth regulatory designation for risvutatug rezetecan, exemplifying the potential of this B7-H3-targeted ADC, which is being developed in a range of solid tumours, including lung, prostate and colorectal cancers.

Read also: GSK pulmonary neuroendocrine carcinoma treatment gets Orphan Drug Designation in EU

Previously, risvutatug rezetecan was granted Priority Medicines (PRIME) Designation by the EMA for relapsed or refractory ES-SCLC and Breakthrough Therapy Designations for relapsed or refractory ES-SCLC and relapsed or refractory osteosarcoma granted by the US FDA.

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