October 19, 2026 - FDA Approves Immunotherapy That May Allow Certain Patients with Rectal Cancer to Avoid Chemotherapy, Radiation and Surgery

The U.S. Food and Drug Administration today granted accelerated approval for a new use of the immunotherapy Jemperli (dostarlimab-gxly) that may allow some patients with locally advanced rectal cancer to avoid common treatments that can have a lasting impact on their daily life.

Patients with locally advanced rectal cancer often receive chemotherapy and radiation prior to surgery, which can affect bowel, urinary, and sexual function, as well as fertility. Surgery also carries risks of serious, sometimes life-threatening complications, including poor healing and leaks where the bowel is reconnected, and in some patients, permanent colostomy.

"The FDA is committed to closing the gap between scientific progress and patient access, and that commitment has to show up not just in policy, but in the pace of our work," said Acting FDA Commissioner Kyle Diamantas, J.D. "American patients deserve timely access to innovative treatments, and the Trump Administration is making that a reality. 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."

Today's approval is for Jemperli used alone in adults with previously untreated stage II or stage III locally advanced rectal cancer. At these stages, the cancer has grown into nearby tissue or reached nearby lymph nodes but has not spread to distant organs. To be eligible, patients' tumors must be mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H), as determined by an FDA-authorized test. Published estimates suggest that approximately 3% of locally advanced rectal cancers are dMMR or MSI-H.

Both terms describe a defect in the way cancer cells correct errors when copying their DNA. Normally, a built-in repair system catches and fixes these mistakes. When the system does not work properly, errors accumulate and create proteins the immune system can recognize, making these tumors particularly vulnerable to immunotherapy.

Jemperli works by blocking a signal that cancer cells use to shield themselves from the immune system. Specifically, it targets a protein called PD-1 on immune cells.

"Standard treatment for rectal cancer has major life-changing effects. For certain types of rectal cancer, this approval represents a groundbreaking option that avoids surgery, radiation and chemotherapy. This represents a significant benefit for patients and their families," said Angelo de Claro, M.D., director of the FDA's Oncology Center of Excellence. "The FDA is acting on emerging evidence to bring new approaches to patients sooner, while requiring further studies to confirm that these treatments provide lasting benefits."

The FDA based its decision on interim results from AZUR-1 (NCT05723562), a phase 2 study evaluating Jemperli alone in 154 patients with previously untreated dMMR or MSI-H locally advanced rectal cancer. In the study, patients received up to nine doses of Jemperli, given every 3 weeks over 6 months. After a median follow-up of more than 2 years - meaning at least half of patients had been monitored for that long - almost three-fourths of patients had not needed surgery, radiotherapy or chemotherapy. Longer follow-up is needed to understand the duration of response and how often the cancer returns.

An earlier study at Memorial Sloan Kettering Cancer Center (NCT04165772) evaluated Jemperli alone in 50 patients with previously untreated dMMR or MSI-H locally advanced rectal cancer. None of the patients had detectable signs of cancer after Jemperli treatment, and nearly all patients (93%) maintained that cancer-free status for at least 12 months. Of the 27 patients followed for at least 3 years, only one had their cancer return, and it recurred in the same area.

Jemperli can cause the immune system to attack healthy organs and tissues. These reactions can be severe or fatal. 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 granted Priority Review to the application and Breakthrough Therapy designation to Jemperli for the treatment of dMMR or MSI-H locally advanced rectal cancer. This action marks the 11th approval of a drug reviewed under the Commissioner's National Priority Voucher pilot program, which aims to help accelerate the review of therapies that address national public health priorities. Because this is an accelerated approval based on interim trial results, the sponsor must continue following patients to show these responses create lasting clinical benefit.

The approval was granted to GSK.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products.

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