Jemperli Approved in the U.S. as First and Only Immunotherapy for Adults with MSI-H/dMMR Locally Advanced Rectal Cancer
AnaptysBio, Inc. a company focused on managing the financial collaborations for Jemperli with Tesaro, a GSK company, and Quimilza (imsidolimab) with Vanda, announced that GSK has received U.S. Food and Drug Administration (FDA) approval for Jemperli (dostarlimab) as the first and only immunotherapy for adults with stage II and III microsatellite instability-high (MSI-H)/ mismatch repair deficient (dMMR) locally advanced rectal cancer.
Jemperli received FDA Fast Track and Breakthrough Therapy designations for this indication. The application was also granted Priority Review and received the National Priority Voucher, resulting in an accelerated approval ahead of the February 2027 PDUFA date.
This approval is supported by data from the AZUR-1 trial. The study met its primary objective, demonstrating a significant proportion of participants achieved sustained clinical complete responses at 12 months (cCR12), with data continuing to mature.
Investigator-assessed data, a clinically relevant endpoint, showed 68% of evaluable patients (98/144; 95% CI: 60%, 76%) achieved cCR12. In an exploratory analysis, 76% (110/144; 95% CI: 69%, 83%) of evaluable patients had no evidence of cancer one year or more after treatment. This includes 68% (98/144) (95% CI: 60%, 76%) of patients who reached cCR12 without surgery, plus other participants (n=12) who had their complete response confirmed through tissue analysis after surgery (pathological complete response, or pCR). No disease recurrences have been reported among responders to date, after a median follow up of more than two years.
Data on the primary objective, cCR12 assessed by independent central review, continue to mature. 47% (54/116) (95% CI: 37%, 56%) of evaluable patients have achieved cCR12. An additional 38 participants who are still progressing through to 12 month follow up have already achieved a clinical complete response.
The safety profile of Jemperli was generally consistent with its established profile across solid tumors. Serious adverse reactions occurred in 20% of patients receiving Jemperli, and the most common adverse reactions were fatigue (32%), rash (22%), diarrhea (18%) and pruritus (17%). Jemperli was permanently discontinued due to adverse reactions in 3% of patients.
GSK plans to present full data from the AZUR-1 interim analysis as a late-breaking abstract at ESMO Congress 2026 in Madrid, Spain on October 25, 2026.
Current standard of treatment for locally advanced rectal cancer includes chemotherapy, radiation and surgery1, but these can have lasting consequences, including bowel, urinary and sexual dysfunction, secondary cancers and infertility.2,3 For patients with MSI-H/dMMR tumors, a biomarker found in around 5-10% of rectal cancers, Jemperli monotherapy could help avoid these interventions.4
The AZUR-1 sBLA is being reviewed under Project Orbis, an FDA Oncology Center of Excellence initiative that enables concurrent review by international health authorities. Regulatory reviews are ongoing in participating countries.
About Jemperli (dostarlimab)
Jemperli is a programmed death receptor-1 (PD-1)-blocking antibody that binds to the PD-1 receptor and blocks its interaction with the PD-1 ligands PD-L1 and PD-L2. In the United States, Jemperli is currently approved for endometrial cancer, rectal cancer and dMMR solid tumors, and is currently being studied as monotherapy in colon cancer and head and neck squamous cell carcinoma.
Jemperli was discovered by AnaptysBio, Inc. and licensed to TESARO, Inc., under a Collaboration and Exclusive License Agreement signed in March 2014. Under this agreement, GSK is conducting ongoing research, development, commercialization, and manufacturing of Jemperli.
More information about the product and its currently approved indications is available at U.S. product information and EU product information.
