TL;DR
The FDA granted accelerated approval to Jemperli (dostarlimab-gxly) alone for adults with untreated stage II or III rectal cancer whose tumors are dMMR or MSI-H. In interim results from a 154-patient study, nearly three-fourths had not needed surgery, radiation or chemotherapy after a median follow-up exceeding two years. Continued follow-up is required to establish how durable the benefit is.
The U.S. Food and Drug Administration on October 19, 2026, granted accelerated approval for Jemperli (dostarlimab-gxly) alone in adults with previously untreated stage II or III locally advanced rectal cancer whose tumors test positive for dMMR or MSI-H. The approval gives some patients an option that may let them avoid chemotherapy, radiation and surgery, but longer follow-up is needed to determine whether the responses last and how often cancer returns.
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The approval applies only to adults with stage II or stage III disease that has not been treated and whose tumors have deficient mismatch repair (dMMR) or high microsatellite instability (MSI-H), identified through an FDA-authorized test. These tumor features involve a fault in the DNA error-repair process. The FDA says published estimates suggest they occur in about 3% of locally advanced rectal cancers.
The FDA based its decision on interim results from AZUR-1, a phase 2 study of 154 patients. Participants received up to nine doses of Jemperli, administered every three weeks over six months. After a median follow-up of more than two years, almost three-fourths had not needed surgery, radiotherapy or chemotherapy, according to the agency. That measure does not by itself establish that the cancer was permanently eliminated.
Jemperli is a type of immunotherapy that blocks PD-1, a protein on immune cells. The drug can prompt the immune system to attack cancer cells, but it can also attack healthy organs and tissues, sometimes causing severe or fatal reactions. The FDA listed fatigue, rash, diarrhea and itching among the most common side effects reported in at least 15% of patients treated for this type of rectal cancer.
A Chance to Avoid Standard Treatments
For eligible patients, the potential change is substantial because the usual treatment pathway can involve chemotherapy and radiation before surgery. The FDA says these treatments can have lasting effects on bowel, urinary and sexual function, as well as fertility. Surgery also carries risks, including poor healing and leaks where the bowel is reconnected; some patients require a permanent colostomy.
The approval does not mean every patient with rectal cancer can skip those treatments. It is limited by stage, prior treatment and tumor biology, and the study results are interim. Patients and their care teams will need to weigh the possibility of avoiding conventional treatment against the known risks of immunotherapy and the remaining uncertainty about long-term cancer control.
In a statement, FDA Oncology Center of Excellence Director Angelo de Claro said standard rectal cancer treatment can have major effects on patients’ lives and called the approval an option for certain tumor types. He also said further studies are needed to confirm lasting benefit.
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Evidence Behind the Accelerated Approval
The agency’s approval is based on interim results from AZUR-1, rather than completed long-term follow-up. The FDA granted the application Priority Review and gave Jemperli Breakthrough Therapy designation for dMMR or MSI-H locally advanced rectal cancer. The agency said the review was completed nearly four months ahead of schedule.
A separate earlier study at Memorial Sloan Kettering Cancer Center examined Jemperli alone in 50 patients with untreated dMMR or MSI-H locally advanced rectal cancer. The FDA reported that none had detectable signs of cancer after treatment and that 93% maintained that status for at least 12 months. Among 27 patients followed for at least three years, one had a recurrence in the same area. These results are from a separate study and should not be combined with AZUR-1’s interim findings.
The FDA described this decision as the 11th approval under the Commissioner’s National Priority Voucher pilot program, which is intended to speed review of therapies addressing national public health priorities. The approval was granted to drugmaker GSK.
“Completing this review nearly 4 months ahead of schedule means some patients who might otherwise be preparing for surgery, radiation or chemotherapy may now have a different conversation with their doctor.”
— Kyle Diamantas, acting FDA commissioner
Durability and Recurrence Still Need Study
The AZUR-1 results are interim, and the FDA says longer follow-up is needed to understand how long responses last and how often cancer returns. The reported finding that nearly three-fourths of participants had not needed surgery, radiation or chemotherapy after a median follow-up of more than two years does not establish that all were cured or that treatment will permanently replace conventional care.
Because the approval is accelerated, GSK must continue following patients to show that the observed responses produce lasting clinical benefit. The announcement does not specify a final completion date for that work. The available information also does not establish how treatment outcomes may vary among patients who meet the eligibility criteria.
Jemperli can cause the immune system to attack healthy tissue, with reactions that the FDA says may be severe or fatal. Patients’ individual risks and treatment decisions require discussion with their oncology team; the approval announcement is not a guarantee that a patient can avoid other treatment.
Follow-Up Data Will Test Lasting Benefit
GSK is required to continue monitoring patients in the confirmatory work tied to the accelerated approval. Longer-term results will help clarify whether responses persist, how frequently disease returns and whether the treatment delivers lasting clinical benefit. The FDA may review those findings as they become available.
For now, the approved use is limited to adults with previously untreated stage II or III locally advanced rectal cancer whose tumors are confirmed as dMMR or MSI-H by an FDA-authorized test. Patients considering Jemperli should discuss the potential benefits, immune-related risks and the possibility of needing additional treatment with their clinicians.
Key Questions
Who is eligible for the FDA-approved use of Jemperli?
The approval covers adults with previously untreated stage II or III locally advanced rectal cancer whose tumors are dMMR or MSI-H, as confirmed by an FDA-authorized test.
Does the approval mean patients can avoid surgery, radiation and chemotherapy?
Some patients may be able to avoid those treatments, but that is not guaranteed. In interim AZUR-1 results, almost three-fourths of participants had not needed surgery, radiotherapy or chemotherapy after a median follow-up of more than two years. Longer follow-up is needed to assess durability and recurrence.
What did the AZUR-1 study report?
The phase 2 study included 154 patients who received up to nine doses of Jemperli over six months. At the interim analysis, almost three-fourths had not needed surgery, radiation or chemotherapy after a median follow-up exceeding two years.
What side effects did the FDA identify?
The most common side effects reported in at least 15% of patients treated for this type of rectal cancer were fatigue, rash, diarrhea and itching. The FDA also warns that Jemperli can cause severe or fatal immune reactions affecting healthy organs and tissues.
Why is more research required after approval?
The approval is accelerated and relies on interim study results. GSK must continue following patients to show that the responses lead to lasting clinical benefit and to provide more information about response duration and cancer recurrence.
Source: primary
