FDA approves Jemperli for certain rectal cancers, allowing many patients to skip surgery and chemo

Overview

Updated 1 hour ago

The FDA approved Jemperli (dostarlimab) on October 22 for adults with a genetic subtype of locally advanced rectal cancer. It is the first immunotherapy cleared for these patients, and trial data show most can skip chemotherapy, radiation, and surgery.

Standard care for this cancer combines chemoradiation with surgery, which can leave lasting bowel, urinary, and sexual problems, plus infertility and secondary cancers. In the 154-patient AZUR-1 trial, 68% of evaluable patients showed no detectable cancer a year after a six-month course of Jemperli infusions, and 71% never needed additional treatment.

Why it matters

For the small subset of rectal cancer patients with MSI-H/dMMR tumors, this could replace chemoradiation and surgery with six months of infusions.

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Key Indicators

68%

12-month sustained clinical complete response (investigator-assessed)

98 of 144 evaluable AZUR-1 patients who received Jemperli alone.

154

Patients in AZUR-1 phase 2 trial

All received up to nine doses of Jemperli over six months.

71%

Patients who needed no surgery, radiation, or chemo after Jemperli

110 of 154 at a median follow-up of 26 months, per the interim analysis.

93%

Cancer-free at 12 months in earlier Memorial Sloan Kettering study

Of 50 patients; 1 of 27 followed for 3 years had the cancer return.

Voices

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Ever wondered what historical figures would say about today's headlines?

People Involved

Anthony Wood

Chief Scientific Officer, GSK

Announced the October 2026 approval

Organizations Involved

U.S. Food and Drug Administration

Federal Regulatory Agency

Granted accelerated approval of Jemperli for rectal cancer

The U.S. agency that approves drugs for the American market; granted Jemperli its first approval in 2021 and its accelerated approval for rectal cancer on October 22, 2026.

GSK (GlaxoSmithKline)

Pharmaceutical Company

Manufacturer of Jemperli; sponsor of the AZUR-1 trial

London-based pharmaceutical company that makes and sells Jemperli (dostarlimab-gxly) and ran the AZUR-1 trial supporting the rectal cancer approval.

Memorial Sloan Kettering Cancer Center

Cancer Research Hospital

Ran the earlier 50-patient study that first showed complete responses

New York cancer center whose earlier study of dostarlimab in 50 rectal cancer patients reported complete responses in nearly all participants.

Timeline

2021 October 2026

3 events Latest: Today

October 2026
FDA grants accelerated approval of Jemperli for rectal cancer
Today Regulatory Approval

First immunotherapy approved for MSI-H/dMMR locally advanced rectal cancer; many patients can avoid surgery and chemoradiation.
Today
April 2026
AZUR-1 interim analysis data cutoff
Trial Data

Of 154 patients, 71% had no further treatment and no progression; 68% showed a 12-month complete response.
April 20th, 2026
2021
FDA approves dostarlimab for endometrial cancer
Regulatory Approval

The drug's first U.S. approval; the PD-1 inhibitor later studied in rectal cancer.
2021

Scenarios

Confirmatory data convert accelerated approval to traditional approval

Possible Resolves by End of 2029

Discussed by: FDA's accelerated approval framework requires verification of clinical benefit; the agency explicitly conditioned approval on continued follow-up

The approval is conditional. GSK must keep tracking AZUR-1 patients and, under the accelerated pathway, must eventually show the responses translate into real, lasting clinical benefit. If follow-up holds, the FDA could lift the conditional status and grant traditional approval for the rectal cancer use.

Long-term data show rare recurrences, cementing Jemperli as an alternative to surgery

Possible Resolves by End of 2027

Discussed by: GSK reported no recurrences among responders after more than two years; Memorial Sloan Kettering saw 1 recurrence in 27 patients followed 3 years

AZUR-1 patients continue under observation. If three-year follow-up shows recurrence stays rare in patients who achieved complete response, Jemperli could consolidate its place as first-line therapy for this biomarker-defined group, potentially reshaping treatment guidelines.

Recurrences emerge with longer follow-up, narrowing Jemperli's role

Unlikely Resolves by End of 2028

Discussed by: The FDA noted longer follow-up is needed to understand how often the cancer returns

The MSK data showed one recurrence among 27 patients at three years. If longer AZUR-1 follow-up reveals a higher recurrence rate, doctors might pair Jemperli with surgery or reserve it for patients who decline or cannot tolerate chemoradiation. The approval itself would not be withdrawn immediately, but clinical use would narrow.

Historical Context

2 moments from history that rhyme with this story — and how they unfolded.

May 2017

Keytruda tumor-agnostic approval (2017)

The FDA approved pembrolizumab (Keytruda) for any solid tumor carrying MSI-H/dMMR mutations, the first time a cancer drug was approved based on a genetic marker rather than the organ where the cancer started.

Then

Opened the door to biomarker-driven approvals across cancer types.

Now

Established the framework Jemperli now rides; MSI-H/dMMR testing became standard in colorectal and other cancers.

Why this matters now

The same MSI-H/dMMR biomarker that first allowed tissue-agnostic approval now determines who is eligible for Jemperli in rectal cancer.

May 2001

Gleevec transforms CML (2001)

The FDA approved imatinib (Gleevec) for chronic myeloid leukemia, a targeted pill that attacked the BCR-ABL protein driving the cancer. Within years it turned a fatal leukemia into a manageable chronic disease.

Then

CML survival rates jumped from months to near-normal lifespans.

Now

Established the paradigm that a drug aimed at a specific molecular driver could replace toxic chemotherapy.

Why this matters now

Gleevec proved a molecularly targeted drug could displace chemotherapy for a defined patient subset. Jemperli is the same idea applied to a small group of rectal cancer patients.