▎WuXiEdited by Kant Content Team

GSK recently announced that the U.S. FDA has granted its PD-1 inhibitor Jemperli (dostarlimab) Breakthrough Therapy Designation (BTD) for the treatment of locally advanced mismatch repair-deficient (dMMR)/high microsatellite instability (MSI-H) rectal cancer. According to the press release,This isdostarlimabThe second regulatory designation obtained for the locally advanced dMMR/MSI-H rectal cancer indication, the therapy in 2Granted Fast Track designation by the FDA in January 2023。

Rectal cancer is a type of colorectal cancer that originates in the rectum and is often classified as part of colorectal cancer.Colorectal cancer is the third most common cancer globally.In the United States, approximately4.610,000 newly diagnosed cases of rectal cancer, of which approximately5-10%Belongs todMMR/MSI-HType.Such tumors are caused byDNAFix functional abnormalities that lead to replication errors.Research shows,dMMRIs a kind of able to predictPD-1Important biomarkers for the efficacy of immune checkpoint inhibition therapy.Tumors with this biomarker are commonly found in endometrial cancer, colorectal cancer, and other gastrointestinal cancers., and may also occur in other types of solid tumors.

Currently, the standard treatment regimen for locally advanced dMMR/MSI-H rectal cancer includes initial chemotherapy combined with radiotherapy, followed by surgical resection of the tumor and part of the intestine or surrounding tissues. Although this therapy can achieve initial efficacy in most patients, nearly one-third of patients eventually die due to cancer metastasis. In addition, surgery and chemoradiotherapy in the standard treatment may have long-term negative impacts on patients' quality of life, such as intestinal, urinary, and sexual dysfunction, as well as secondary cancers and infertility issues.

The granting of this Breakthrough Therapy Designation is primarily based on a study conducted byGSKSupported and collaborated with Memorial Sloan Kettering Cancer Center on2Preliminary data from Phase clinical trials.TestTest shows,Accept and completeDostarlimab asFirst-line therapy42 namesLocally AdvanceddMMRPatients with rectal cancer,Achieved an unprecedented 100% clinical complete response (cCR) rate, meaning no tumor was detected through magnetic resonance imaging (MRI), endoscopy, positron emission tomography (PET), or digital rectal examination (DRE).In the first batch24Among the patients, the median follow-up time was26.3Months (95% CI：12.4-50.5) when persistentcCR。

The safety and tolerability of Dostarlimab are generally consistent with its known safety profile. No grade 3 or higher adverse events were reported in the trial. The trial is still ongoing to further evaluate the efficacy in patients.

[1] Jemperli (dostarlimab) receives US FDA Breakthrough Therapy Designation for locally advanced dMMR/MSI-H rectal cancer. Retrieved December 18, 2024 from https://www.gsk.com/en-gb/media/press-releases/jemperli-dostarlimab-receives-us-fda-breakthrough-therapy-designation-for-locally-advanced-dmmrmsi-h-rectal-cancer/

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