GSK旗下Jemperli获FDA加速批准，成为MSI-H/dMMR局部晚期直肠癌首款免疫疗法

GSK plc (GSK) announced that the U.S. FDA has approved Jemperli as the first and only immunotherapy for adults with stage II and III microsatellite instability-high (MSI-H) or mismatch repair-deficient (dMMR) locally advanced rectal cancer. The approval was granted under the FDA's accelerated approval pathway and is based on results from the Phase II AZUR-1 trial.
Offering an Alternative to Chemotherapy, Radiation and Surgery
Treatment for locally advanced rectal cancer has traditionally involved a combination of chemotherapy, radiation therapy and surgery. While these treatments can be effective, they may also lead to long-term complications affecting bowel, urinary and sexual function.
According to GSK, Jemperli monotherapy may offer an alternative treatment approach for patients whose tumors can carry MSI-H/dMMR biomarkers, which are found in approximately 5% to 10% of rectal cancers.
AZUR-1 Trial Results
The FDA approval was supported by data from the Phase II AZUR-1 study, which evaluated Jemperli as a stand-alone treatment in previously untreated patients with stage II and III MSI-H/dMMR locally advanced rectal cancer.
Investigator-assessed data showed that 68% of evaluable patients (98 of 144) achieved a sustained clinical complete response at 12 months, meaning no detectable signs of cancer were found without the need for surgery. In an exploratory analysis, 76% of evaluable patients had no evidence of cancer one year or more after treatment, including patients whose complete responses were confirmed through surgery-related tissue analysis.
The company reported that no disease recurrences have been observed among responders after a median follow-up of more than two years.
Safety Profile
GSK said the safety profile of Jemperli was generally consistent with previous studies across solid tumors. Serious adverse reactions occurred in 20% of patients. The most commonly reported side effects were fatigue, rash and diarrhea, and itching. Treatment was permanently discontinued due to adverse events in 3% of patients.
About Jemperli and Next Steps
Jemperli (dostarlimab) is a PD-1 blocking immunotherapy designed to help the body's immune system recognize and attack cancer cells. The drug is already approved for certain gynecological cancers and is being studies across multiple tumor types, including colorectal, head and neck and other gastrointestinal cancers.
The AZUR-1 study enrolled 154 patients who received nine cycles of Jemperli over six months. The trial was designed to assess whether the therapy could enable patients to avoid chemotherapy, radiation and surgery while maintaining durable cancer control.
GSK said full interim results from AZUR-1 will be presented at the European Society for Medical Oncology (ESMO) Congress in October 2026. Regulatory reviews of the application are also ongoing in the multiple countries through the FDA-led Project Orbis initiative.
GSK has traded between $42.85 and $61.70 over the past year. The stock closed Monday's trading at $49.46, down 0.10%.
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October 16, 2026 16:06 ET Monthly consumer price inflation data and retail sales figures were the main news from the U.S. economy this week. In Europe, monthly GDP figures from the U.K. gained attention. External trade data from China was in focus in Asia. Elsewhere, the International Monetary Fund released its latest projections for the global economy.