GSK’s Jemperli accepted for priority FDA review in dMMR/MSI‑H locally advanced rectal cancer
- FDA assigned a PDUFA action date of February 2027 for the Jemperli sBLA.
- AZUR‑1, a registrational phase II single‑arm trial (n=154), met its primary endpoint demonstrating sustained clinical complete responses for 12 months (cCR12).
- AZUR‑1 dosing: nine cycles over six months, dostarlimab 500 mg IV every three weeks; interim safety consistent with its known profile.
- The application is accepted under Project Orbis and is eligible for expedited review via the National Priority Voucher program.
Regulatory status
The US FDA has accepted for priority review a supplemental Biologics License Application for dostarlimab (Jemperli) in previously untreated stage II and III mismatch repair‑deficient (dMMR)/MSI‑high (MSI‑H) locally advanced rectal cancer, with a PDUFA action date in February 2027. The filing is eligible for expedited review through the National Priority Voucher program and has been accepted under Project Orbis; regulatory decisions remain independent in each country.
AZUR‑1 clinical data
The submission is supported by registrational phase II, single‑arm AZUR‑1 data (n=154) that met its primary objective by demonstrating a meaningful and sustained clinical complete response rate for 12 months (cCR12) with no detectable signs of cancer for at least one year. Interim safety and tolerability were generally consistent with dostarlimab’s well‑characterised profile across solid tumours. These data are scheduled for presentation at a scientific congress later in 2026.
Trial design and dosing
AZUR‑1 evaluated dostarlimab monotherapy in previously untreated stage II/III dMMR/MSI‑H locally advanced rectal cancer. Patients received nine cycles of dostarlimab over six months, administered as 500 mg intravenous infusions every three weeks.
Clinical context and implications
Rectal cancer affects around 770,000 people globally each year and approximately 5–10% of cases are dMMR/MSI‑H. Current standard of care—chemotherapy, radiation and surgery—can have lasting effects on bowel, urinary and sexual function, fertility and quality of life. AZUR‑1 results build on earlier research showing clinical complete responses without other treatments and support the potential for dostarlimab, if approved, to eliminate or delay the need for chemo, radiation and surgery for some patients in this population; dostarlimab is not currently approved for locally advanced rectal cancer.
Source: GSK