GSK Plc (LON:GSK) today announced the US Food and Drug Administration (FDA) has accepted for priority review a supplemental Biologics License Application (sBLA) for Jemperli (dostarlimab) for patients with previously untreated stage II and III mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) locally advanced rectal cancer. 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 result in an earlier FDA decision1.
Rectal cancer, a type of bowel cancer, affects around 770,000 people globally each year2 and approximately 5-10% of cases have the dMMR/MSI-H subtype3. Chemotherapy, radiation and surgery are the standard of care and while often effective, they can have lasting adverse effects on bowel, urinary and sexual function, fertility and overall quality of life4,5,6.
The application is based on positive data from the registrational phase II, single-arm AZUR-1 trial, which 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. In interim data, the safety and tolerability profile of dostarlimab was generally consistent with its well-characterised and manageable safety profile observed across solid tumours. These data will be submitted for presentation at a scientific congress later in 2026.
AZUR-1 results represent a substantial improvement compared to the historical standard of care7. The data support the potential for dostarlimab, if approved, to become the first immunotherapy capable of eliminating or delaying the need for chemotherapy, radiation and surgery for some patients in this population. These findings build on earlier research conducted with Memorial Sloan Kettering Cancer Center (MSK), which first demonstrated the potential for dostarlimab to achieve clinical complete responses without other treatments in patients with dMMR/MSI-H locally advanced rectal cancer.
Dostarlimab was previously granted Fast Track and Breakthrough Therapy Designations in this setting8,9. The application has also been accepted under Project Orbis, an FDA Oncology Center of Excellence initiative that enables coordinated reviews by international health authorities and may support earlier regulatory decisions and patient access. Regulatory decisions remain independent in each country.
References
1. FDA press release issued 6 November, 2025. FDA Awards Second Batch of National Priority Vouchers. Available at https://www.fda.gov/news-events/press-announcements/fda-awards-second-batch-national-priority-vouchers
2. International Agency for Research on Cancer (IARC) (2024) Rectum cancer fact sheet: GLOBOCAN 2022. World Health Organization. Available at: https://gco.iarc.who.int/media/globocan/factsheets/cancers/9-rectum-fact-sheet.pdf. Accessed August 2026
3. Cercek A, et al. Mismatch Repair-Deficient Rectal Cancer and Resistance to Neoadjuvant Chemotherapy. Clin Cancer Res. 2020 Jul 1;26(13):3271-3279. doi: 1158/1078-0432.CCR-19-3728. Epub 2020 Mar 6. PMID: 32144135; PMCID: PMC7348681
4. Cercek, A., Lumish, M., Sinopoli, J. et al. (2022) ‘PD‑1 blockade in mismatch repair-deficient, locally advanced rectal cancer’, New England Journal of Medicine, 386(25), pp. 2363-2376. Available at: https://www.nejm.org/doi/full/10.1056/NEJMoa2201445
5. Negro, S. et al. (2025) ‘Quality of life in rectal cancer treatments: a systematic review’, Cancers, 17(14), 2310. Available at: https://www.mdpi.com/2072-6694/17/14/2310
6. Neibart, S.S. et al. (2020) ‘Quality of life after radiotherapy for rectal cancer’, Current Colorectal Cancer Reports, 16(1), pp. 1-10. Available at: https://pmc.ncbi.nlm.nih.gov/articles/PMC7336840/
7. Cercek A, Roxburgh CS, Strombom P, et al. Adoption of Total Neoadjuvant Therapy for Locally Advanced Rectal Cancer. JAMA Oncol. 2018;4(6):e180071. doi:10.1001/jamaoncol.2018.0071
8. GSK press release issued 16 December 2024. Jemperli (dostarlimab) receives US FDA Breakthrough Therapy Designation for locally advanced dMMR/MSI-H rectal cancer. Available at https://www.gsk.com/en-gb/media/press-releases/jemperli-dostarlimab-receives-us-fda-breakthrough-therapy-designation-for-locally-advanced-dmmrmsi-h-rectal-cancer/
9. GSK press release issued 9 February 2023. US FDA Advisory Committee votes in support of trials designed to evaluate Jemperli (dostarlimab-gxly) as a potential treatment for mismatch repair-deficient/microsatellite instability-high locally advanced rectal cancer. Available at https://www.gsk.com/en-gb/media/press-releases/us-fda-advisory-committee-votes-in-support-of-trials-designed-to-evaluate-jemperli-dostarlimab-gxly-as-a-potential-treatment-for-dmmrmsi-h-locally-advanced-rectal-cancer/
10. Andre T, Berton D, Curigliano G, et al. Antitumor Activity and Safety of Dostarlimab Monotherapy in Patients With Mismatch Repair Deficient Solid Tumors: A Nonrandomized Controlled Trial. JAMA Netw Open. 2023;6(11):e2341165. doi:10.1001/jamanetworkopen.2023.41165.
11. Le DT, et al. PD-1 blockade in tumors with mismatch repair deficiency. N Engl J Med. 2015;372(26):2509-2520.
12. National Cancer Institute at the National Institutes of Health. Definition of mismatch repair deficiency. Accessed August 2026. Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms/def/mismatch-repair-deficiency
13. JEMPERLI (dostarlimab) Summary of Product Characteristics. GlaxoSmithKline (Ireland) Limited. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/jemperli. Accessed August 2026.
14. JEMPERLI (dostarlimab-gxly) prescribing information. GlaxoSmithKline. Available at: https://gskpro.com/content/dam/global/hcpportal/en_US/Prescribing_Information/Jemperli/pdf/JEMPERLI-PI-MG.PDF/. Accessed August 2026.
