FDA grants priority review to GSK’s Jemperli for dMMR/MSI-H locally advanced rectal cancer

GSK plc has recently announced that the U.S. FDA has accepted for priority review a supplemental Biologics License Application for Jemperli (dostarlimab), intended for patients with previously untreated stage II and III mismatch repair deficient (dMMR) or microsatellite instability high (MSI-H) locally advanced rectal cancer. The FDA has set a PDUFA action date of February 2027. The application also qualifies for expedited review under the National Priority Voucher program, which could lead to an earlier decision from the agency.

Rectal cancer, a form of bowel cancer, affects roughly 770,000 people worldwide each year, with an estimated 5 to 10 percent of cases falling into the dMMR/MSI-H subtype. Standard treatment typically involves chemotherapy, radiation, and surgery, and while these approaches are often effective, they can leave lasting effects on bowel, urinary, and sexual function, fertility, and overall quality of life.

The application draws on positive results from the registrational Phase II, single arm AZUR-1 trial, which met its primary objective by showing a meaningful and sustained 12 month clinical complete response rate, meaning no detectable signs of cancer for at least a year. In interim data, dostarlimab’s safety and tolerability profile was largely consistent with the well characterized, manageable profile observed across other solid tumor types. These findings are expected to be submitted for presentation at a scientific conference later in 2026.

The AZUR-1 results mark a substantial improvement over the historical standard of care. They support the possibility that dostarlimab, if approved, could become the first immunotherapy able to eliminate or delay the need for chemotherapy, radiation, and surgery for some patients in this group. These findings build on earlier work conducted with Memorial Sloan Kettering Cancer Center, which first showed that dostarlimab could produce clinical complete responses without additional treatment in patients with dMMR/MSI-H locally advanced rectal cancer.

Dostarlimab had already received Fast Track and Breakthrough Therapy designations in this setting. The application has also been accepted under Project Orbis, an initiative run by the FDA’s Oncology Center of Excellence that allows international health authorities to conduct coordinated reviews, potentially enabling earlier regulatory decisions and patient access, though each country’s regulatory decision remains independent.

---

---

Related Topics and Keywords

dMMR/MSI-H locally advanced rectal cancer, FDA, GSK, JEMPERLI, rectal cancer