GSK's Jemperli Receives US FDA Breakthrough Therapy Designation for Rectal Cancer

GSK plc has received Breakthrough Therapy Designation from the US Food and Drug Administration (FDA) for Jemperli (dostarlimab), an immunotherapy drug, for the treatment of patients with locally advanced mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) rectal cancer.

The designation is based on preliminary clinical evidence showing a 100% clinical complete response rate in all 42 patients who completed treatment with dostarlimab in a GSK-supported collaborative study. A clinical complete response means no evidence of disease was found.

This designation is granted to drugs that have the potential to show improvement over available therapies for serious conditions. The current standard of care for this patient group, which involves chemotherapy, radiation, and surgery, can lead to significant long-term quality-of-life issues.

The data supporting the designation comes from an ongoing phase II study in collaboration with Memorial Sloan Kettering Cancer Center. GSK's ongoing AZUR-1 trial aims to confirm these findings.

Dostarlimab is not yet approved anywhere globally for the frontline treatment of locally advanced dMMR/MSI-H rectal cancer.