RegulationAccelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan), Orphan Drug (Australia), Priority Review (Australia), Priority Review (United States)

Structure/Sequence

Sequence Code 12981416H

Source: *****

Sequence Code 13030670L

Source: *****

Clinical Trials associated with Retifanlimab

NCT07219576

/ RecruitingPhase 1/2IIT

PRISM: Phase 1b of Retifanlimab and Ruxolitinib In Solid Malignancies Progressing on Prior Checkpoint Inhibition

The goal of this clinical trial is to learn what dose of ruxolitinib can be given safely together with retifanlimab in patients with metastatic renal cell carcinoma and non-small cell lung carcinoma.
The main question it aims to answer is:
What is the maximum dose of ruxolitinib that can be used safely in patients with metastatic renal cell carcinoma and non-small cell lung carcinoma, and will it work?
Participants will:
Take drug ruxolitinib twice a day and keep a diary of when they take ruxolitinib at home; visit the clinic for infusions of retifanlimab every 4 weeks; visit the clinic for checkups and tests.

Start Date08 Jan 2026

Sponsor / Collaborator

University of California San Diego

[+1]

NCT06896188

/ RecruitingPhase 1IIT

A Phase Ib Study of 9-ING-41 (Elraglusib), a Glycogen Synthase Kinase 3-beta (GSK-3β) Inhibitor, Combined With Retifanlimab, a PD-1 Inhibitor, Plus Modified FOLFIRINOX as Frontline Therapy for Patients With Advanced Pancreatic Adenocarcinoma (RiLEY)

This is a study of the combination of 9 ING-41 (elraglusib) and retifanlimab plus mFOLFIRINOX in patients with pancreatic cancer without prior systemic therapy for advanced disease. The safety lead-in cohort will consist of 6 patients, followed by dose de-escalation if necessary, based on safety assessments. After evaluating the safety and tolerability at the initial dose level, the study will proceed to an expansion cohort at the determined safe dose level, with the total maximum enrollment not exceeding 12 patients for the entire study.

Start Date22 Sep 2025

Sponsor / Collaborator

University of Pittsburgh

[+2]

NCT06959537

/ RecruitingPhase 1IIT

A Phase Ib Study of Novel Combination (New) of Low Dose Oral CyclophoSphamide (s) to Potentiate Axatilimab (A) + Retifanlimab (R) in Treating Metastatic Triple Negative Breast Cancer (TNBC)

The goal of this clinical research study is to find the best dose of the drugs cyclophosphamide and axatilimab that can be given in combination with the standard dose of retifanlimab to patients with metastatic TNBC.

Start Date13 Aug 2025

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

100 Clinical Results associated with Retifanlimab

100 Translational Medicine associated with Retifanlimab

100 Patents (Medical) associated with Retifanlimab

Literatures (Medical) associated with Retifanlimab

01 Dec 2025·Lancet Respiratory Medicine

Retifanlimab versus placebo in combination with platinum-based chemotherapy in patients with first-line non-squamous or squamous metastatic non-small-cell lung cancer (POD1UM-304): a phase 3, multiregional, placebo-controlled, double-blind, randomised study

Article

Author: Gogishvili, Miranda ; Munteanu, Mihaela C ; Cornfeld, Mark ; Shi, Jianhua ; Moiseenko, Fedor ; Kuchava, Vladimer ; Cicin, Irfan ; Cil, Timucin ; Nguyen, Hoang Gia ; Arslan, Cagatay ; Lu, Shun ; Vynnychenko, Oleksandr ; Petrovic, Marina ; Nguyen, Duong Thanh ; Kulyaba, Yaroslav ; Artac, Mehmet ; Ibrahim, Aishah ; Sette, Claudia V M ; Bibichadze, Khatuna ; Olmez, Omer Fatih ; Bondarenko, Igor ; Tian, Chuan

BACKGROUND:

Checkpoint inhibitors, including combinations with standard-of-care chemotherapy, have shown survival benefit in patients with metastatic non-small-cell lung cancer (NSCLC); however, access to these drugs varies. We aimed to evaluate the efficacy of the PD-1 inhibitor retifanlimab plus platinum-based chemotherapy as first-line treatment for non-squamous or squamous metastatic NSCLC.

METHODS:

POD1UM-304 was a phase 3, multiregional, placebo-controlled, double-blind, randomised study conducted in approximately 124 hospitals and private clinical centres in 16 countries. Male and female adults aged 18 years or older with squamous or non-squamous stage IV NSCLC (staging by American Joint Committee on Cancer version 8) with Eastern Cooperative Oncology Group performance status 0 or 1 and no previous systemic therapy for metastatic NSCLC were eligible. Patients were randomly assigned (2:1) using interactive response technology to receive intravenous retifanlimab 375 mg or matching placebo on day 1 of each 21-day cycle plus standard platinum-based chemotherapy according to tumour histology for up to 2 years. Patients with non-squamous NSCLC received pemetrexed 500 mg/m² plus cisplatin 75 mg/m² on day 1 for four cycles, or carboplatin area under the curve (AUC) 5 on day 1 for four cycles, followed by pemetrexed 500 mg/m² on day 1 of each subsequent 21-day cycle, all administered intravenously, until disease progression or unacceptable toxicity. Patients with squamous NSCLC received intravenous carboplatin AUC 6 plus intravenous paclitaxel 200 mg/m² on day 1 for four cycles or intravenous nab-paclitaxel 100 mg/m² on days 1, 8, and 15 for four cycles. Treatment with retifanlimab or placebo was given for up to 35 cycles, unless there was disease progression, unacceptable toxicity, or withdrawal of consent. Randomisation was stratified by PD-L1 expression tumour proportion score, geographical region, and predominant tumour histology. The primary endpoint was overall survival, defined as time from randomisation until death due to any cause, analysed in the full analysis set. Safety was evaluated in all randomly assigned patients who received at least one dose of study drug. This trial is registered with ClinicalTrials.gov (NCT04205812) and is active but no longer enrolling.

FINDINGS:

Between Sept 11, 2020, and March 14, 2023, 1388 patients were assessed for eligibility, 583 of whom were randomly assigned to retifanlimab plus chemotherapy (n=391) or placebo plus chemotherapy (n=192). 381 (65%) patients had non-squamous NSCLC and 202 (35%) had squamous NSCLC; 467 (80%) patients were male and 116 (20%) were female. Median age was 64 years (IQR 58-68). Median overall survival was longer in the retifanlimab plus chemotherapy group than in the placebo plus chemotherapy group (18·1 months [95% CI 16·2-21·0] vs 13·4 months [11·0-16·7]; hazard ratio 0·75 [95% CI 0·60-0·93]; p=0·0042). Overall, higher incidences of treatment-emergent adverse events that were serious (158 [41%] of 389 vs 57 [30%] of 190), grade 3 or worse (238 [61%] vs 103 [54%]), or led to retifanlimab or placebo dose delay (169 [43%] vs 67 [35%]) or discontinuation (33 [8%] vs nine [5%]) were observed in the retifanlimab plus chemotherapy group than in the placebo plus chemotherapy group. The proportions of fatal COVID-19-related treatment-emergent adverse events were similar in the retifanlimab plus chemotherapy group and the placebo plus chemotherapy group (four [1%] vs five [3%]).

INTERPRETATION:

Retifanlimab improved overall survival compared with placebo when added to platinum-based chemotherapy, with a safety profile that is representative of previous PD-1 and PD-L1 inhibitor-chemotherapy combinations. Adding retifanlimab to first-line chemotherapy could be a potential treatment option for patients with squamous or non-squamous metastatic NSCLC.

FUNDING:

Incyte.

13 Aug 2025·Immunotherapy

Successful treatment of localized Merkel cell carcinoma with avelumab in a patient with amyotrophic lateral sclerosis

Article

Author: Tomsitz, Dirk ; Leding, Julia ; French, Lars E. ; Janjic, Nina ; Arnold, Lisa ; Buchillon, Raquel ; Senner, Sonja ; Frey, Surina ; Heinzerling, Lucie

Currently, the first-line treatment of non-metastatic Merkel cell carcinoma (MCC) is complete resection. In case of unresectable or metastatic MCC, immune checkpoint inhibitor (ICI) therapy with avelumab (or in the US also pembrolizumab or retifanlimab) is indicated. We report on a patient with a primary, non-metastatic MCC on the left eyelid and amyotrophic lateral sclerosis (ALS). Due to ALS, the patient's communication was limited to eye movement and blinking. Complete resection or definitive radiotherapy of the tumor while preserving the function of the eye muscles was not possible. No prior data was available for patients with ALS under ICI therapy. In agreement with the patient and his family, a monotherapy with avelumab, a programmed death ligand 1 (PD-L1) inhibitor, was initiated. This led to a complete remission of the tumor with a progression-free survival of over 24 months and importantly no deterioration of the ALS.

01 Aug 2025·Journal for ImmunoTherapy of Cancer

Phase II study of retifanlimab in patients with recurrent locally advanced or metastatic Merkel cell carcinoma (POD1UM-201)

Article

Author: Montaudié, Henri ; Gaudy-Marqueste, Caroline ; Bhatia, Shailender ; Cornfeld, Mark ; Spada, Francesca ; Grignani, Giovanni ; Morano, Federica ; Rutkowski, Piotr ; Guida, Michele ; Burgess, Melissa ; Pulini, Jennifer ; Yeung, Cecilia C S ; Depenni, Roberta ; Spagnolo, Francesco ; Tian, Chuan ; Lebbé, Céleste

Background:

POD1UM-201, an open-label, single-arm, phase II multiregional study, evaluated efficacy and tolerability of retifanlimab, a humanized monoclonal antibody targeting programmed cell death protein-1 (PD-1) in chemotherapy-naive patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC).

Methods:

Patients were enrolled across 34 sites in the USA, Canada, and Europe. Eligible patients were ≥18 years with confirmed recurrent advanced locoregional or metastatic MCC not amenable to surgery or radiation therapy, had not received previous systemic treatment, had measurable disease, and Eastern Cooperative Oncology Group performance status 0–1. Retifanlimab 500 mg was administered intravenously every 4 weeks (Q4W) for up to 2 years. The primary endpoint was objective response rate (ORR). Key secondary endpoints included duration of response (DOR), disease control rate (DCR), progression-free survival (PFS), overall survival (OS), and safety.

Results:

A total of 101 patients were enrolled between February 12, 2019, and June 16, 2021, with a median duration of follow-up for response of 22.2 (range: 1.1–55.3) months. ORR was 54.5% (95% CI: 44.2% to 64.4%; n=55), including 18 patients (17.8%) with complete responses (CRs) and 37 (36.6%) with partial responses (PRs). DCR was 60.4% (95% CI: 50.2% to 70.0%; n=61). Median DOR was not reached (95% CI: 14.0 months to not estimable (NE)) in patients who achieved CR and was 25.3 months (95% CI: 14.2 months to NE) in those with PR. With a median follow-up of 9.3 (range: 0.0–57.1) months, the estimated median PFS was 16.0 months (95% CI: 9.0 months to 32.2). Median OS was not reached with 63% of patients alive at 3 years. The safety profile was representative of the PD-(ligand)1 inhibitor class, with grade 3 immune-related adverse events occurring in 11 patients (10.9%).

Conclusions:

Retifanlimab 500 mg Q4W led to frequent and durable responses in chemotherapy-naive patients with advanced MCC with an acceptable safety profile. Retifanlimab represents a new immunotherapy option for patients with locally advanced or metastatic MCC.

Trial registration number:

NCT03599713; EudraCT 2018-001627-39

123

News (Medical) associated with Retifanlimab

30 Jan 2026

·www.businesswire.com

Incyte Announces Positive CHMP Opinion for Zynyz ® (retifanlimab) for First-Line Treatment of Advanced Squamous Cell Carcinoma of the Anal Canal (SCAC)

MORGES, Switzerland--(BUSINESS WIRE)--Incyte (Nasdaq:INCY) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending the approval of Zynyz® (retifanlimab) in combination with carboplatin and paclitaxel (platinum-based chemotherapy) for the first-line treatment of adult patients with metastatic or with inoperable locally recurrent squamous cell carcinoma of the anal canal (SCAC). "Today’s positive CHMP opinion is an important step towards addressing the urgent need for new treatment options for patients in Europe with advanced SCAC, a disease which has seen limited innovation for decades,” said Lee Heeson, Head Incyte Intnl. “Today’s positive CHMP opinion is an important step towards addressing the urgent need for new treatment options for patients in Europe with advanced SCAC, a disease which has seen limited innovation for decades,” said Lee Heeson, Executive Vice President and Head of Incyte International. “If approved, Zynyz in combination with platinum-based chemotherapy has the potential to become a new standard-of-care for patients living with this rare and difficult-to-treat cancer.” The positive CHMP opinion was based on data from the Phase 3 POD1UM-303/InterAACT2 trial evaluating Zynyz in combination with (platinum-based chemotherapy) in adult patients with metastatic or inoperable locally recurrent SCAC not previously treated with systemic chemotherapy.5 Results from POD1UM-303/InterAACT2 (NCT04472429) published in The Lancet showed a statistically significant 37% reduction in the risk of progression or death (P=0.0006).4 Patients in the Zynyz and (platinum-based chemotherapy) combination group achieved a median progression-free survival (PFS) of 9.3 months compared to 7.4 months for patients in the placebo combination group.4 No new safety signals were identified. Serious adverse reactions occurred in 47% of patients receiving Zynyz in combination with chemotherapy.4 The most frequent serious adverse reactions (≥ 2% of patients) were sepsis, pulmonary embolism, diarrhoea and vomiting.4 SCAC is the most common type of anal cancer, accounting for approximately 85% of all anal cancer cases.6 Globally, the prevalence of SCAC is estimated at around 1 or 2 cases per 100,000 people, with a higher incidence in women than in men.1,2,3 The CHMP opinion is now being reviewed by the European Commission, which has the authority to grant approval for all centrally authorized products in the EU. About Squamous Cell Carcinoma of the Anal Canal (SCAC) Worldwide, SCAC is the most common type of anal cancer, making up 85% of cases.6 It is a rare disease for which the incidence increases approximately 3% per year.7,8,9,10 About 90% of cases are associated with human papillomavirus (HPV) infection—the number one risk factor for anal cancer.10 HIV is an important amplifier of anal cancer, as people with HIV are 25 to 35 times more likely to develop it.11,12 Anal cancer shares many of the same symptoms as non-cancerous conditions, such as hemorrhoids—including pain, itching, a lump or mass and changes in bowel movements—and as a result can go undetected leading to the majority of patients presenting with locally advanced disease.13 About POD1UM The POD1UM (PD1 Clinical Program in Multiple Malignancies) clinical trial program for retifanlimab includes POD1UM-303, POD1UM-202 and several other Phase 1, 2 and 3 studies for patients with solid tumors. For more information about the study, please visit https://clinicaltrials.gov/study/NCT04472429. About Zynyz® (retifanlimab) Zynyz® (retifanlimab) is a humanized monoclonal antibody targeting programmed death receptor-1 (PD-1), indicated in combination with carboplatin and paclitaxel (platinum-based chemotherapy) for the first-line treatment of adult patients with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC) in the U.S. and Japan and as a single agent for the treatment of adult patients with locally recurrent or metastatic SCAC with disease progression or intolerance to platinum-based chemotherapy in the U.S. Zynyz is also indicated as monotherapy for the first-line treatment of adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC) in the U.S, EU, Canada, and Switzerland. Zynyz is marketed by Incyte in the U.S. In 2017, Incyte entered into an exclusive collaboration and license agreement with MacroGenics, Inc. for global rights to retifanlimab. Zynyz is a registered trademark of Incyte. For more information, see the Zynyz SmPC. About Incyte A global biopharmaceutical company on a mission to Solve On, Incyte follows science to find solutions for patients with unmet medical needs. Through the discovery, development, and commercialization of proprietary therapeutics, Incyte has established a portfolio of first-in-class medicines for patients and a strong pipeline of products in Hematology, Oncology and Inflammation & Autoimmunity. Headquartered in Wilmington, Delaware, Incyte has operations in North America, Europe, and Asia. For additional information on Incyte, please visit Incyte.com or follow us on social media: LinkedIn, X, Instagram, Facebook, YouTube. Forward-Looking Statements Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding whether and when Zynyz might provide a successful treatment option or become the standard-of-care for patients in Europe with advanced SCAC, contain predictions, estimates, and other forward-looking statements. These forward-looking statements are based on Incyte's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the European Commission, the U.S. FDA and other regulatory authorities; the efficacy or safety of Incyte’s partners' products; the acceptance of Incyte and its partners' products in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; and other risks detailed from time to time in Incyte's reports filed with the Securities and Exchange Commission, including its annual report on Form 10-K and its report on Form 10-Q for the quarter ended September 30, 2025. Incyte disclaims any intent or obligation to update these forward-looking statements. 1 National Cancer Institute. Cancer stat facts: anal cancer. Available at: https://seer.cancer.gov/statfacts/html/anus.html. Accessed January 2026 2 Mignozzi S., Santucci C., Malvezzi M., et al. (2024) Global trends in anal cancer incidence and mortality. Eur J Cancer Prev, 33:77-86. Link to source (https://pubmed.ncbi.nlm.nih.gov/38047709/) 3 English K.J. (2024) Anal carcinoma - exploring the epidemiology, risk factors, pathophysiology, diagnosis, and treatment. World J Exp Med, 14:98525. Link to source (https://pubmed.ncbi.nlm.nih.gov/39312693/) 4 Rao S., Samalin-Scalzi E., Evesque L., et al. (2025) Retifanlimab with carboplatin and paclitaxel for locally recurrent or metastatic squamous cell carcinoma of the anal canal (POD1UM-303/InterAACT-2): a global, phase 3 randomised controlled trial. Lancet, 405(10495):2144-52. Link to source (https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(25)00631-2/fulltext) 5 Clinical Trials.Gov. Carboplatin-paclitaxel with retifanlimab or placebo in participants with locally advanced or metastatic squamous cell anal carcinoma (POD1UM-303/InterAACT 2). Available at: https://clinicaltrials.gov/study/NCT04472429. Accessed January 2026 6 Symer M.M., Yeo H.L. (2018) Recent advances in the management of anal cancer. F1000Research, 7:F1000 Faculty Rev-1572. Link to source (https://pubmed.ncbi.nlm.nih.gov/30345012/) 7 Islami F., Ferlay J., Lortet-Tieulent J., et al. (2017) International trends in anal cancer incidence rates. Int J Epidemiol, 46:924–938. Link to source (https://pubmed.ncbi.nlm.nih.gov/27789668/) 8 Giuliano A.R., Nyitray A.G., Kreimer A.R., et al. (2015) EUROGIN 2014 roadmap: differences in human papillomavirus infection natural history, transmission and human papillomavirus-related cancer incidence by gender and anatomic site of infection. Int J Cancer, 136:2752-2760. Link to source (https://pubmed.ncbi.nlm.nih.gov/25043222/) 9 Morris V., Eng C. (2016) Strengthening the immunotherapy paradigm in anal cancer. Available at: https://c.peerview.com/live/programs/150210387-1/downloads/PVI_slides_SCAC-SF25.pdf?ProjectNumber=150210387_1. Accessed Janaury 2026 10 U.S. Centers for Disease Control and Prevention. Cancers linked with HPV each year. Available at: https://www.cdc.gov/cancer/hpv/cases.html. Accessed January 2026 11 Wang C.C.J., Sparano J., Palefsky J.M. (2017) Human immunodeficiency virus/AIDS, human papillomavirus, and anal cancer. Surg Oncol Clin N Am, 26:17-31. Link to source (https://pubmed.ncbi.nlm.nih.gov/27889034/) 12 NCCN clinical practice guidelines in oncology: cancer in people with HIV. Version 1.2021. 2021 13 Anal Cancer Foundation. Anal cancer: signs, symptoms, causes & treatment. Available at: https://www.analcancerfoundation.org/what-is-anal-cancer/. Accessed January 2026

Phase 3Clinical ResultLicense out/inDrug ApprovalImmunotherapy

30 Jan 2026

·endpoints.news

CHMP opposes Lilly’s Mounjaro in heart failure, backs Novo’s Kayshild for liver disease

EU regulators have refused to greenlight Eli Lilly’s weight loss drug Mounjaro as a treatment for a type of heart failure, while giving the go-ahead for Novo Nordisk’s semaglutide in liver disease. Lilly failed to get support from the European Medicines Agency’s human medicines committee, or CHMP, on Friday for Mounjaro to treat chronic heart failure with preserved ejection fraction in adults with obesity. HFpEF is when the heart becomes too stiff to pump enough blood around the body. The committee said Mounjaro didn’t reduce the number of patient deaths due to heart problems in patients with obesity and HFpEF. While the drug did reduce the number of heart failure-related hospitalizations, it’s unclear whether that was due to the weight loss-independent effect of Mounjaro, CHMP said. Lilly had also been angling after the heart failure label expansion in the US, but withdrew the application for Mounjaro — marketed as Zepbound for weight loss — from the FDA in May. Despite declining to expand Mounjaro’s label for heart failure, the committee said it would include the data from Lilly’s Phase 3 SUMMIT study in the drug’s product information. Meanwhile, Novo Nordisk won the panel’s go-ahead for semaglutide, branded as Kayshild, to be used as a treatment for patients with non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with liver fibrosis stages F2 to F3. Semaglutide is already available in the US to treat patients with MASH, after securing FDA approval in August. CHMP also gave positive opinions for four other new therapies: The agency also gave recommendations to extend the labels of nine therapies:

Drug ApprovalPhase 3Accelerated Approval

30 Jan 2026

·firstwordpharma.com

CHMP backs Novo's Kayshild for MASH, reverses course on Sanofi's Rezurock

The EU's Committee for Medicinal Products for Human Use (CHMP) closed January with a slate of decisions Friday, including recommendations for Novo Nordisk and UCB drugs, while Sanofi's graft-versus-host disease (GvHD) treatment gets a second change and Eli Lilly settles for a label update.Novo Nordisk's KayshildOne of the more closely watched decisions was the conditional recommendation for Novo Nordisk's semaglutide to treat non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) and liver fibrosis, a new indication for the GLP-1 agonist that the company plans to market as Kayshild. Semaglutide is already approved for diabetes as Ozempic and Rybelsus, and as Wegovy for weight loss.The opinion marks the first time a GLP-1 agonist has been cleared in Europe for MASH, and comes about five months after US regulators approved it under the Wegovy brand for the same indication based on data from the Phase III ESSENCE trial. Results there showed that 37% of patients achieved improved liver scarring without their steatohepatitis getting worse at 72 weeks, compared to 22.5% on placebo. Rates of steatohepatitis resolution without any increase in liver scarring were 62.9% and 34.1%, respectively.Sanofi's Rezurock re-examinedAfter having revisited its October negative opinion of Sanofi's Rezurock (belumosudil) for chronic GvHD, the EU drug advisory panel now recommends conditional approval in last-line patients ages 12 years and up. The ROCK2 inhibitor downregulates IL-17 and IL-21 and mediates signalling in immune cellular function and fibrotic pathways.Sanofi picked up the oral ROCK2 inhibitor via its $1.9-billion Kadmon buyout in 2021, just after the FDA had cleared it for GvHD. In the pivotal Phase II ROCKstar study, the drug plus standard of care delivered about a 73% response rate in patients who had received two to five prior lines of systemic therapy, with a median duration of 1.9 months.At the time, CHMP said ROCKstar's single-arm design made it hard to clearly determine Rezurock's benefit, while Sanofi said it wouldn't have confirmatory data until 2030. Still, the French drugmaker requested a re-examination of the negative October decision, and said Friday's CHMP recommendation was based on safety and efficacy data "from several clinical studies and real-world evidence," including ROCKstar."We…made the commitment to conduct a new post-approval confirmatory study, given the limited late-line treatment options available for EU patients," said Olivier Charmeil, Sanofi's EVP of general medicines.UCB's KygevviCHMP also issued a positive opinion under exceptional circumstances for Kygevvi, UCB's treatment for thymidine kinase 2 deficiency, a rare and often fatal mitochondrial myopathy. It is indicated for patients whose disease started at or before 12 years of age. Kygevvi, which was approved by the FDA last November, works by incorporating the pyrimidine nucleosides, deoxycytidine and deoxythymidine, into skeletal muscle cell mitochondria to help rebuild their DNA.The EU recommendation relied on a retrospective chart review and a single-arm Phase II study in 39 children, in which 84% regained one or more motor milestones after treatment with Kygevvi, suggesting improved motor function. The most common side effects were diarrhoea, vomiting and abdominal pain.A recommendation under exceptional circumstances means the authorisation can be granted with specific obligations that are reviewed each year.Incyte's Zynyz expandedIn oncology, CHMP backed an extension for Incyte's PD-1–blocking antibody Zynyz (retifanlimab) to treat adults with inoperable, metastatic or locally recurrent squamous cell carcinoma of the anal canal (SCAC) when combined with chemotherapy. The drug was initially authorised in Europe in 2024 for Merkel cell carcinoma.US regulators greenlit Zynyz for SCAC last year based on results from the Phase III POD1UM-303 trial. According to CHMP, half of SCAC patients treated with Zynyz plus chemotherapy lived for 9.3 months or longer without their cancer worsening, versus 7.4 months for placebo and chemotherapy.However, regulators acknowledged lingering uncertainty over overall survival benefit; half of patients treated with Zynyz lived for at least 32.8 months compared with 22.2 months for placebo, which was not statistically significant. Still, the committee pointed to improvements in progression-free survival as sufficient evidence of clinical value in a setting with few alternatives.Lilly's Mounjaro for heart failureMeanwhile, not all GLP-1 news ended with CHMP backing. The EU panel declined to grant a separate heart-failure indication for Lilly's dual GLP-1/GIP agonist Mounjaro (tirzepatide) in patients with obesity and preserved ejection fraction (HFpEF), instead opting to incorporate trial results into the product label.The drug is currently approved in Europe for treating type 2 diabetes and for chronic weight management in adults. "Although EMA did not recommend that a separate indication should be granted for the treatment of HFpEF, it agreed to include relevant data from the study submitted with the application in the medicine's product information," CHMP said, so that "healthcare professionals have access to up-to-date data on the effects of Mounjaro in adults with chronic HFpEF and obesity."Lilly withdrew a similar application last year in the US, where tirzepatide is sold as Zepbound for weight loss, after the FDA asked to see more clinical data. The filing was based on Phase III data from the SUMMIT study showing that Zepbound reduced the risk of heart failure complications by 38% versus placebo in patients with and without diabetes.

Clinical ResultPhase 3Drug ApprovalPhase 2

100 Deals associated with Retifanlimab

External Link

KEGG	Wiki	ATC	Drug Bank
D11827	-	-	DB15766

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Anal canal squamous cell carcinoma	United States	Incyte Corp.	15 May 2025
Metastatic Merkel Cell Carcinoma	United States	Incyte Corp.	22 Mar 2023

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
squamous cell carcinoma of the anus	NDA/BLA	European Union	Incyte Biosciences Distribution BV	29 Jan 2026
Endometrial Carcinoma	Phase 3	China	Zai Lab (Shanghai) Co., Ltd.	20 Sep 2021
metastatic non-small cell lung cancer	Phase 3	China	Incyte Corp.	18 Sep 2020
metastatic non-small cell lung cancer	Phase 3	China	Zai Lab (Shanghai) Co., Ltd.	18 Sep 2020
Metastatic Non-Squamous Non-Small Cell Lung Carcinoma	Phase 3	United States	Incyte Corp.	11 Sep 2020
Metastatic Non-Squamous Non-Small Cell Lung Carcinoma	Phase 3	China	Incyte Corp.	11 Sep 2020
Metastatic Non-Squamous Non-Small Cell Lung Carcinoma	Phase 3	Brazil	Incyte Corp.	11 Sep 2020
Metastatic Non-Squamous Non-Small Cell Lung Carcinoma	Phase 3	Bulgaria	Incyte Corp.	11 Sep 2020
Metastatic Non-Squamous Non-Small Cell Lung Carcinoma	Phase 3	Czechia	Incyte Corp.	11 Sep 2020
Metastatic Non-Squamous Non-Small Cell Lung Carcinoma	Phase 3	Hungary	Incyte Corp.	11 Sep 2020

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04472429 (POD1UM-303, ESMO_IO2025)	Phase 3	squamous cell carcinoma of the anus First line	308	Retifanlimab + carboplatin-paclitaxel	iipyfmvixu(jgahhharik) = bdodguyxxr gdwvcccist (xymrrwjxsb ) View more	Positive	12 Nov 2025
				Placebo + carboplatin-paclitaxel	iipyfmvixu(jgahhharik) = ldojjtflqq gdwvcccist (xymrrwjxsb ) View more
EUCTR2021-005775-39-BE (ESMO2025)	Phase 2	PD-L1+	176	Retifanlimab (RETI)	nlttgtuuzy(bbqpplrzqr) = ioutssdkqf tutanjfviw (uavvpnlpdh, 3.5 - 9.7) View more	Negative	17 Oct 2025
				Retifanlimab + Tuparstobart (doublet tx)	nlttgtuuzy(bbqpplrzqr) = ebzkzmcpkj tutanjfviw (uavvpnlpdh, 3.7 - 7.6) View more
NCT04205812 (POD1UM-304, Pubmed) Manual	Phase 3	Non-Small Cell Lung Cancer First line	583	Retifanlimab + chemotherapy	zmpxjprkal(axxqsrviyn) = jnuezkgkct auhoqmmsbx (cmouxzkcxc, 16.2 - 21.0) View more	Positive	19 Sep 2025
				Placebo + chemotherapy	zmpxjprkal(axxqsrviyn) = lxpulkbnse auhoqmmsbx (cmouxzkcxc, 11.0 - 16.7) View more
NCT03532295 (CTIM-23, CTgov)	Phase 2	Glioblastoma Multiforme \| Glioblastoma \| Recurrent Glioblastoma	51	Radiation therapy+Bevacizumab+Retifanlimab (Regimen A: Retifanlimab+RT+bevacizumab)	cfdoaljnkt = dkfkmbkyrt qrjrnonaym (zfifbbscvd, zfnwbisevq - ozxoujfqmv) View more	-	07 Aug 2025
				Radiation therapy+Bevacizumab+Epacadostat+Retifanlimab (Regimen B: Retifanlimab+RT+bevacizumab+epacadostat)	cfdoaljnkt = bnxtylcntu qrjrnonaym (zfifbbscvd, piuymnvogz - eshoiqtwzt) View more
NCT04116073 (CTgov)	Phase 2	Pancreatic carcinoma non-resectable	25	PD-1 antibody+INCMGA00012	imimavidva = zfcivtleah tgojbhjcpe (vudohsvhoy, ixjqysmliu - cszcikzeoa) View more	-	29 Jul 2025
NCT05239182 (RiLEY, CTgov)	Phase 2	Pancreatic adenocarcinoma \| Pancreatic Ductal Adenocarcinoma \| Advanced Pancreatic Adenocarcinoma	7	9-ING-41+Abraxane+Gemcitabine+Retifanlimab	tfhjqfdjsv = trbyxpublu dcxrrhmxlc (vzthoncxsz, rltxklzjgr - qbgyllplun) View more	-	17 Jul 2025
NCT04472429 (POD1UM-303/InterAACT 2, BusinessWire) Manual	Phase 3	Anal canal squamous cell carcinoma	308	Retifanlimab + Carboplatin + Paclitaxel	osnlulgtyq(pkcepkmept) = xbwcwvpzcm tnpmpvuppl (cqgkbdkrej, 7.5 - 11.3) View more	Positive	14 Jun 2025
				Placebo + Carboplatin + Paclitaxel	osnlulgtyq(pkcepkmept) = iwrhixjcbr tnpmpvuppl (cqgkbdkrej, 7.1 - 7.7) View more
NCT04472429 (POD1UM-303/InterAACT 2, ASCO2025) Manual	Phase 3	Anal canal squamous cell carcinoma	308	Retifanlimab + chemotherapy (PD-L1 ≥ 1%)	krmglulmkl(fuqxthcbfy) = eumbjjoygk kqywpvwlbq (tcwebfhhdn ) View more	Positive	30 May 2025
				Retifanlimab + chemotherapy (PD-L1 < 1%)	krmglulmkl(fuqxthcbfy) = dblmlbswmg kqywpvwlbq (tcwebfhhdn )
PODIUM303/InterAACT 2 (ASCO2025)	Phase 3	Anal canal squamous cell carcinoma First line	-	Carboplatin + paclitaxel	xhwtkvfcic(yqauxrwian) = xydwdyofqi dhqbpjahqx (vttbkyrunn ) View more	Positive	30 May 2025
				Immune checkpoint inhibitors (ICIs)	mjwmxicmqo(gzjafdhkkp) = xviqfozykt mevxpbwmrt (ihanryggvz, 5.5 - 23.0)
NCT03599713 (POD1UM-201, ASCO2025)	Phase 2	Metastatic Merkel Cell Carcinoma	101	Retifanlimab 500 mg	seksrunume(swoubrdaol) = mwesjowlml dvthbydtlb (mroqeytkmg ) View more	Positive	30 May 2025

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