The NHS has made no UK-wide decision on dostarlimab rectal cancer treatment, as of 20 October 2026. The US Food and Drug Administration (FDA) granted accelerated approval on 19 October 2026. The product is GSK’s Jemperli (dostarlimab). Wales has supported off-label use since August 2023. NICE lists an appraisal as awaiting development, with no publication date.
What did the FDA approve on 19 October 2026?
The FDA granted accelerated approval on 19 October 2026. The indication covers adults with stage II or III locally advanced rectal cancer. The tumour must be microsatellite instability high (MSI-H) or mismatch repair deficient (dMMR). An FDA-authorised test must confirm this.
The recommended dose is 500 mg intravenously every three weeks, for up to nine cycles. The dostarlimab rectal cancer approval came four months ahead of the FDA goal date, the agency says. GSK had earlier given a February 2027 action date.
The review used priority review, breakthrough therapy designation and Real-Time Oncology Review. It also used the Commissioner’s National Priority Voucher pilot. Accelerated approval means further studies must confirm lasting benefit. The FDA page does not name the confirmatory requirements.
What did the AZUR-1 trial show?
The dostarlimab rectal cancer indication rests on AZUR-1 (NCT05723562). It is a multicentre, single-arm, open-label phase 2 trial. The FDA counts 154 treated patients, all previously untreated.
The primary endpoint was a clinical complete response sustained for 12 months (cCR12). An independent central review assessed it. That review found cCR12 in 47 per cent of evaluable patients (54 of 116).
GSK’s approval release gives a 95 per cent confidence interval of 37 to 56 per cent. The FDA page prints the lower bound as 27 per cent. The two sources differ, and GSK’s figure is used here.
Investigators reported a higher rate of 68 per cent (98 of 144). The 95 per cent confidence interval was 60 to 76 per cent. The two assessments used different evaluable groups, and 116 is fewer than the 154 treated. GSK says another 38 patients had a clinical complete response but under 12 months of follow-up. The company adds that data are still maturing.
At data cutoff, 110 patients (71 per cent) had received dostarlimab alone without progression. Another 26 (17 per cent) had surgery. Thirteen (8 per cent) had other systemic therapy or radiation.
Median follow-up was 26.3 months, according to the FDA. It reports no progression among patients with cCR12, a clinical complete response or a near complete response.
The supporting study
The FDA also reviewed NCT04165772, an investigator-led study of 50 patients. It reported cCR12 in 93 per cent (95 per cent confidence interval 82 to 99 per cent). Of 27 patients followed for at least three years, one had a local recurrence. That patient was treated with lymphadenectomy.
Neither study had a comparator arm. GSK says full interim AZUR-1 data will come at the European Society for Medical Oncology (ESMO) Congress. That meeting runs from 23 to 27 October 2026 in Madrid. GSK lists AZUR-1 as late-breaking abstract LBA87, due on 25 October.
Dr Andrea Cercek is AZUR-1 principal investigator and section head of colorectal cancer at Memorial Sloan Kettering Cancer Center. In GSK’s release she said the results “demonstrate the potential for a fundamentally different treatment approach”.
What does the approval not show?
The evidence is single-arm. No randomised comparison with standard chemoradiotherapy and surgery exists in the FDA notice. Investigators and the independent review also gave different response rates.
The FDA page read on 20 October 2026 reports no overall survival results. The AZUR-1 figures are interim, and GSK says the data are still maturing. A confirmatory study will therefore matter for the long-term answer.
The label is also tied to a test. Patients must have MSI-H or dMMR disease, as determined by an authorised test. The approval does not cover rectal cancers without that biomarker. Any NHS decision would need to define the same population.
What are the safety findings?
GSK describes the safety profile as generally consistent with dostarlimab’s established profile across solid tumours. It reports serious adverse reactions in 20 per cent of patients. The most common reactions were fatigue (32 per cent) and rash (22 per cent). Diarrhoea (18 per cent) and pruritus (17 per cent) followed. Three per cent of patients permanently discontinued because of adverse reactions.
The FDA notice lists label warnings. They cover immune-mediated reactions, infusion-related reactions, complications of allogeneic stem cell transplantation and embryo-fetal toxicity. It gives no incidence rates.
A July 2025 Welsh safety alert flagged bowel obstruction from tumour narrowing during treatment. It advises clinicians to watch for new gastrointestinal symptoms after the first cycle.
Dostarlimab rectal cancer: what is the UK status today?
Licence status
The UK summary of product characteristics on the emc was revised on 25 September 2026. It lists two endometrial cancer indications. A check on 20 October 2026 found no rectal cancer indication.
The FDA lists the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) as a Project Orbis partner. It names Health Canada, Israel’s Ministry of Health and Swissmedic as the other partners, with reviews ongoing. A web search on 20 October 2026 found no published MHRA decision on this use.
Wales
The only UK-nation decision on dostarlimab rectal cancer comes from Wales. The One Wales Medicines Process supported dostarlimab for first-line treatment of stage II or III dMMR rectal cancer. The decision was issued in August 2023.
The page labels it an interim decision. The group retained the advice in October 2024. It says dostarlimab is not licensed for this use, so prescribers must secure informed consent.
Support applies only at the approved commercial arrangement price. Patients must be registered with a Welsh GP practice. One course of up to nine cycles is allowed. The page gives a review after two years, and the evidence report cites October 2026.
England and Scotland
NICE lists the topic as ID6747. Its status is awaiting development, and the expected publication date is to be confirmed. The page gives no referral date or remit wording.
A search found no Scottish Medicines Consortium advice on this use. GSK sponsors a retrospective chart review (IRAS 344563) of patients treated through compassionate use or the One Wales route. The HRA summary lists participants from Europe, Asia and South America. It gives a four-year UK study duration but names no participating centres.
NICE faces a similar sequence on another dMMR cancer. An earlier article covers atezolizumab for dMMR colon cancer, which is in NICE scoping.
What would settle the NHS access question?
The Welsh decision shows one route for dostarlimab rectal cancer. It also shows the limits. The decision is interim, and it applies only to patients registered with a Welsh GP. Four events would clarify access elsewhere. The first is the ESMO presentation of full AZUR-1 data on 25 October 2026. The second is the Welsh review, due around October 2026 according to the evidence report. The third is an MHRA decision, which has no public date. The fourth is the start of the NICE appraisal.
Testing also matters. Michael Sapienza is chief executive of the Colorectal Cancer Alliance. In GSK’s release he said biomarker testing at diagnosis “will be essential to identifying patients who may be eligible”.
For patients outside Wales, the answer is not yet known. Dostarlimab rectal cancer access depends on an MHRA decision and a NICE appraisal that has not started. Pending US decisions are tracked on the FDA drug approval decisions tracker.
