LONDON - GSK plc (LSE/NYSE: GSK) has announced that Jemperli (dostarlimab) has received Breakthrough Therapy Designation from the US Food and Drug Administration (FDA) for the treatment of patients with locally advanced mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) rectal cancer. The designation is based on clinical data demonstrating a 100% clinical complete response rate in all 42 patients who completed treatment in an ongoing phase II trial.
The FDA's Breakthrough Therapy Designation is intended to expedite the development and review of drugs that may offer significant improvements over existing therapies for serious conditions. Dostarlimab, a programmed death receptor-1 (PD-1)-blocking antibody, has previously received Fast Track designation for the same patient population in January 2023.
The current standard of care for dMMR/MSI-H locally advanced rectal cancer involves chemotherapy, radiation, and surgery, which can lead to long-term adverse effects on quality of life. The promising results from the phase II trial suggest dostarlimab could potentially change the treatment paradigm for these patients.
The trial, conducted in collaboration with Memorial Sloan Kettering Cancer Center, showed that all patients who completed treatment with dostarlimab had no evidence of disease, as assessed by various medical imaging and examination techniques. A sustained clinical complete response was observed in the first 24 patients evaluated, with a median follow-up of 26.3 months. The treatment's safety profile was consistent with known information, with no grade 3 or higher adverse events reported.
While Jemperli is not yet approved for the treatment of locally advanced dMMR/MSI-H rectal cancer, it is currently indicated in the US for certain patients with advanced or recurrent endometrial cancer and for those with dMMR recurrent or advanced solid tumors under accelerated approval.
GSK's ongoing phase II registrational AZUR-1 trial aims to confirm the findings from the current study. The company is also exploring Jemperli in a robust clinical trial program for various cancers, including gynecologic, colorectal, and lung cancers.
This breakthrough designation underscores the potential of dostarlimab in providing a new therapeutic option for patients with locally advanced dMMR/MSI-H rectal cancer, a subset of colorectal cancer that is particularly challenging to treat. The information for this article is based on a press release statement from GSK.
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