By VETTAPHARMA reporter – Derek Roche: The U.S. Food and Drug Administration (FDA) has accepted GSK’s supplemental Biologics License Application (sBLA) for Jemperli (dostarlimab) for previously untreated adults with stage II and III mismatch repair-deficient (dMMR)/microsatellite instability-high (MSI-H) locally advanced rectal cancer, granting the application Priority Review.
The FDA has assigned a PDUFA action date of February 2027. The application is also eligible for expedited review through the National Priority Voucher Program, which could potentially result in an earlier regulatory decision.
The application is supported by data from the registrational Phase 2 AZUR-1 trial, a global, open-label, single-arm study involving 154 patients with previously untreated stage II and III dMMR/MSI-H locally advanced rectal cancer. Patients received nine cycles of Jemperli over six months, administered as a 500 mg intravenous infusion every three weeks.
The trial was designed to evaluate whether Jemperli monotherapy could produce a sustained clinical complete response for at least 12 months (cCR12) and potentially allow some patients to avoid chemotherapy, radiation, and surgery.
According to interim data, the trial met its primary objective by demonstrating a meaningful and sustained clinical complete response rate at 12 months, with no detectable signs of cancer for at least one year in a significant proportion of participants. GSK said the safety and tolerability profile was generally consistent with the established safety profile of Jemperli across solid tumors.
If approved, Jemperli could become the first immunotherapy capable of eliminating or delaying the need for chemotherapy, radiation, and surgery for some patients with this specific form of locally advanced rectal cancer.
Approximately 5–10% of rectal cancers are estimated to have the dMMR/MSI-H subtype. These tumors have defects in their ability to repair DNA damage, resulting in an accumulation of mutations that can make them particularly responsive to immunotherapy.
Jemperli has previously received Fast Track and Breakthrough Therapy Designations from the FDA for this setting. The application has also been accepted under Project Orbis, an FDA Oncology Center of Excellence initiative that facilitates coordinated regulatory reviews among participating international health authorities. Regulatory decisions remain independent in each country.
Jemperli was originally discovered by AnaptysBio and licensed to TESARO, which was subsequently acquired by GSK. GSK is responsible for the ongoing research, development, commercialization, and manufacturing of Jemperli. The medicine is not currently approved anywhere in the world for locally advanced rectal cancer.
Quick FAQs
1. What Is Jemperli?
Jemperli (dostarlimab) is a programmed death receptor-1 (PD-1)-blocking antibody developed by GSK and being investigated across multiple cancer types.
2. What Happened?
The FDA accepted GSK’s application for Priority Review for Jemperli in previously untreated stage II and III dMMR/MSI-H locally advanced rectal cancer.
3. What Study Supports The Application?
The application is supported by the Phase 2 AZUR-1 trial, a global, open-label, single-arm registrational study involving 154 patients.
4. What Were The Key Findings?
AZUR-1 met its primary objective by demonstrating a meaningful and sustained clinical complete response at 12 months, with no detectable signs of cancer for at least one year in a significant proportion of participants.
5. What Could Jemperli Mean For Patients?
If approved for this indication, Jemperli could potentially allow some patients to avoid or delay chemotherapy, radiation, and surgery, potentially reducing treatment-related effects on bowel, urinary, and sexual function, fertility, and quality of life.
6. What Happens Next?
The FDA has assigned a PDUFA action date of February 2027. The application is also eligible for expedited review through the National Priority Voucher Program and has been accepted under Project Orbis.
