FDA Approves Dostarlimab in Previously Untreated Locally Advanced Rectal Cancer

The FDA has granted accelerated approval to dostarlimab-gxly (Jemperli) for the treatment of patients with previously untreated stage II or stage III locally advanced mismatch repair deficient (dMMR) or microsatellite instability–high (MSI-H) rectal cancer, according to a press release from the FDA.¹ The approval is the eleventh to come through the Commissioner’s National Priority Voucher pilot program.

Supporting data for the approval came from the phase 2 AZUR-1 trial (NCT05723562), which evaluated dostarlimab in the indicated patient population. Previously, based on findings from AZUR-1, the FDA granted priority review to the supplemental biologics license application for dostarlimab in this indication. A Prescription Drug User Fee Act action date was set for February 2027.²

In AZUR-1, with a median follow-up of more than 2 years, almost three-fourths of patients had not needed surgery, chemotherapy, or radiotherapy. Further results from AZUR-1 will be presented at the 2026 European Society for Medical Oncology Congress.

In an earlier phase 2 trial (NCT04165772), dostarlimab demonstrated a meaningful and sustained clinical complete response at 12 months, with no signs of cancer detected after dostarlimab treatment, and 93% maintaining that cancer-free status for at least 12 months.

“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,” stated Angelo de Claro, MD, director of the FDA’s Oncology Center of Excellence, in the press release.¹ “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 AZUR-1 trial enrolled 154 patients with previosuly untreated dMMR/MSI-H locally advanced rectal cancer who received up to 9 doses of dostarlimab given every 3 weeks over 6 months.

Notably, dostarlimab may cause the immune system to attack healthy organs and tissues, and the most common adverse effects include fatigue, rash, diarrhea, and itching.

“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,” acting FDA Commissioner Kyle Diamantas, JD, said in the press release. “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.”

References

1. FDA approves immunotherapy that may allow certain patients with rectal cancer to avoid chemotherapy, radiation and surgery. News release. FDA. October 19, 2026. Accessed October 19, 2026. https://tinyurl.com/yj2k5zh4
2. Jemperli (dostarlimab) accepted for priority review by the US FDA for dMMR/MSI-H locally advanced rectal cancer. News release. GSK. August 24, 2026. Accessed October 19, 2026. https://tinyurl.com/2p9nwv9z