Cabozantinib Ipsen® (cabozantinib) accepted by Scottish Medicines Consortium for adults with advanced neuroendocrine tumours

- 10 August 2026 - 10 mins read
  • Cabozantinib Ipsen® (cabozantinib) is accepted by the Scottish Medicines Consortium (SMC) for restricted use within NHS Scotland for the treatment of adult patients with unresectable or metastatic, well-differentiated extra-pancreatic (epNET) and pancreatic (pNET) neuroendocrine tumours who have progressed following at least one prior systemic therapy other than somatostatin analogues, for use when best supportive care would otherwise be considered1
  • Approval for use across NHS Scotland is based on the pivotal CABINET Phase III trial which demonstrated a 77% (hazard ratio [HR] 0.23; 95% confidence interval [CI] 0.12-0.42; p<0.001) and 62% (HR 0.38; 95% CI 0.25-0.59; p<0.001) reduction in the risk of disease progression or death versus placebo for pNETs and epNETs respectively. The median progression-free survival (PFS) for cabozantinib was 13.8 months versus 4.4 months for placebo in the advanced pNETs cohort, while the PFS was 8.4 months for cabozantinib versus 3.9 for placebo in the epNETs cohort2
  • NETs affect approximately 7 per 100,000 people in Scotland,3 and treatment options following disease progression are often limited4,5

LONDON, U.K., 10 August 2026 – Ipsen U.K. (Euronext: IPN; ADR: IPSEY) announced today that cabozantinib is accepted by the SMC for restricted use within NHS Scotland for the treatment of adult patients with unresectable or metastatic, well-differentiated extra-pancreatic (epNET) and pancreatic (pNET) neuroendocrine tumours who have progressed following at least one prior systemic therapy other than somatostatin analogues, for use when best supportive care would otherwise be considered.1

“This recommendation by the SMC is a positive and welcome development for people living with advanced neuroendocrine tumours (NETs) in Scotland,” commented Professor Nicholas Reed, Consultant Clinical Oncologist at the Beatson Oncology Centre, Glasgow. “The availability of an additional treatment option on the NHS in Scotland provides greater disease control for people living with NETs, helping them stay well for as long as possible. As an oral treatment, this may also reduce the need for patients to spend time in hospital, enabling them to spend more quality time with their families and caregivers.”

NETs are rare, with a prevalence of 7 per 100,000 people in Scotland.3 They typically develop slowly, and people living with NETs may require multiple lines of therapy as the disease progresses.4,6,7 Treatment options following progression are often limited and can vary depending on the primary tumour site and other factors, making it challenging to define the optimal sequencing of treatments.4,5

“Neuroendocrine cancers can have a profound impact on people affected by the disease and those closest to them,” commented Lisa Walker, CEO of Neuroendocrine Cancer UK. “Neuroendocrine tumours (NETs), a type of neuroendocrine cancer, often grow at a different and frequently slower rate than many other cancers, including neuroendocrine carcinomas (NECs). As a result, symptoms may not appear in the earliest stages or may be mistaken for those of more common conditions, meaning diagnosis is often delayed until the disease is more advanced. The symptoms can be debilitating and life-limiting, placing a significant physical and emotional burden on patients and their families. The availability of an additional treatment option for people living with NETs through NHS Scotland is therefore an important development, offering greater choice, renewed hope, and the potential to improve outcomes for those affected by this form of neuroendocrine cancer.”

The SMC decision was based on data from the Phase III CABINET trial which investigated cabozantinib versus placebo in people living with advanced pNETS and epNETs who have progressed following at least one prior systemic therapy other than somatostatin analogues. The primary endpoint in each cohort was (PFS), defined as the time from randomisation to radiographic progressive disease, according to RECIST 1.1, determined retrospectively by blinded independent central review (BICR), or death from any cause.2  Key secondary endpoints included overall survival, objective response and safety.2 The trial demonstrated a 77% and 62% reduction in the risk of disease progression or death versus placebo for pNETs (hazard ratio [HR] 0.23; 95% confidence interval [CI] 0.12–0.42; p<0.001) and epNETs (HR 0.38; 95% CI 0.25–0.59; p<0.001), respectively.2

In the pNET cohort (n=95), at a median follow-up of 13.8 months, median PFS was 13.8 months for patients treated with cabozantinib (n=64) versus 4.4 months in the placebo group (n=31) (stratified HR for disease progression or death 0.23; 95% CI 0.12-0.42; p<0.001).2 For patients with epNETs (n=203), at a median follow-up of 10.2 months, median PFS was 8.4 months in the cabozantinib group (n=134) versus 3.9 months for the placebo group (n=69) (stratified HR for disease progression or death 0.38; 95% CI 0.25–0.59; p<0.001).2 Overall health-related quality of life remained stable over time and was similar in the two groups among patients who completed questionnaires.2 No overall survival difference between the trial groups has been observed to date.2

“We are pleased that cabozantinib has been accepted by the SMC for eligible adults with NETs in Scotland,” said Dr Ian Gray, U.K. & Ireland Senior Medical and Regulatory Affairs Director at Ipsen. “This decision marks an important milestone for the NETs community and reflects Ipsen’s continued commitment to bringing additional treatment options to people living with rare and difficult-to-treat cancers.”

The safety profile of cabozantinib observed in the CABINET trial was consistent with its known safety profile and no new safety signals were identified. Grade 3 or higher adverse events were noted in 41 (65%) of the patients with pNETs and 82 (62%) of the patients with epNETs treated with cabozantinib compared with 7 (23%) and 18 (27%) of the patients, respectively, who received placebo.2 Common treatment-related adverse events of Grade 3 or higher included hypertension, fatigue, diarrhoea and thromboembolic events.2 Grade 5 adverse events occurred in 9 patients (7%) in the cabozantinib group and 4 patients (6%) in the placebo group among those with epNETs, while no Grade 5 events were reported among patients with pNETs.2

Cabozantinib is currently undergoing review by the National Institute for Health and Care Excellence (NICE) in this indication. Ipsen will continue to work closely with NICE and NHS England and remains committed to supporting access for eligible NETs patients across England and Wales.

About Cabozantinib Ipsen® (cabozantinib)1

The detailed recommendations for the use of cabozantinib are described in the Summary of Product Characteristics (SmPC) from the Medicines and Healthcare products Regulatory Agency (MHRA).

Exelixis granted Ipsen U.K. exclusive rights for the commercialisation and further clinical development of cabozantinib outside of the U.S. and Japan.

In over 65 countries outside of the U.S. and Japan, including in the European Union, cabozantinib also has indications in advanced renal cell carcinoma, locally advanced or metastatic differentiated thyroid carcinoma, and hepatocellular carcinoma.

About the Phase III CABINET trial2

The multicentre Phase III CABINET (randomised, double-blinded study of CABozantinib versus placebo In patients with advanced NEuroendocrine Tumours after progression following prior systemic therapy other than SSAs) trial enrolled 298 patients in the U.S. at the time of the analysis.

Patients were randomised 2:1 to cabozantinib or placebo in two separately powered cohorts: pNETS and epNETs. The epNETs cohort included patients with the following primary tumour sites: gastrointestinal (GI) tract, lung, thymus, unknown primary and other organs. Each cohort was randomised separately and had its own statistical analysis plan. Patients must have had measurable disease per RECIST 1.1 criteria and must have experienced disease progression or intolerance after at least one U.S. Food and Drug Administration-approved line of prior systemic therapy other than SSAs. The primary endpoint in each cohort was PFS per RECIST 1.1 by retrospective blinded independent central review, or death from any cause. Upon confirmation of disease progression, patients were unblinded, and those receiving placebo were permitted to cross over to open-label therapy with cabozantinib. Secondary endpoints included overall survival, objective response rate and safety. More information about this trial is available at ClinicalTrials.gov.

About NETs 

NETs are a group of tumours that develop in the cells of the neuroendocrine system throughout the body.2,6 The most common sites of NETs include the gastrointestinal (GI) tract, lungs, and pancreas.8,9

The symptoms of NETs are often not distinct and difficult to identify, leading to delays in diagnosis, with 58% of patients presenting with metastatic disease at diagnosis.10 Based on the Surveillance, Epidemiology, and End Results (SEER) data, the 5-year relative survival rate for patients with advanced GI NETs and lung NETs is approximately 68% and 49%, respectively.11,12

NETs affect approximately 7 per 100,000 people in Scotland.3 The number of people newly diagnosed with NETs globally is believed to be rising, with a higher estimated prevalence than pancreatic or bladder cancer.12,13,14

About Ipsen

We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. Our pipeline is fuelled by internal and external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France, and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries.

Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit https://www.ipsen.com/uk-ireland/.

Ipsen Contacts

Media (Local)

Sam Howland | sam.howland@ipsen.com

Fi Warren  | fi.warren@envisionpharma.com

Tom Clare-Ducler  | tom.clare-ducler@envisionpharma.com

Disclaimers and/or forward-looking statements

The forward-looking statements, objectives and targets contained herein are based on Ipsen’s management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect Ipsen’s future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words ‘believes’, ‘anticipates’ and ‘expects’ and similar expressions are intended to identify forward-looking statements, including Ipsen’s expectations regarding future events, including regulatory filings and determinations. Moreover, the targets described in this document were prepared without taking into account external-growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by Ipsen. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising medicine in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. Ipsen must face or might face competition from generic medicine that might translate into a loss of market share. Furthermore, the research and development process involves several stages each of which involves the substantial risk that Ipsen may fail to achieve its objectives and be forced to abandon its efforts with regards to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and healthcare legislation and risks arising from unexpected regulatory or political changes such as changes in tax regulation and regulations on trade and tariffs, such as protectionist measures, especially in the United States; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen’s patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen’s activities and financial results. Ipsen cannot be certain that its partners will fulfil their obligations. It might be unable to obtain any benefit from those agreements. A default by any of Ipsen’s partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen’s business, financial position or performance. Ipsen expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. Ipsen’s business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to Ipsen’s latest Universal Registration Document, available on ipsen.com.

  1. Summary of Product Characteristics. Cabozantinib Ipsen. Available upon request.
  2. Chan JA et al. Phase 3 Trial of Cabozantinib to Treat Advanced Neuroendocrine Tumors. New England Journal of Medicine. 2024;392[7]:653-665.
  3. Scottish Neuroendocrine Tumour Group (SCONET). Consensus Guidelines for the Management of Patients with Neuroendocrine Tumours. Originally published: 2015; updated July 2021. Available from: https://www.woscan.scot.nhs.uk/wp-content/uploads/SCONET-Guideline-April-2022-v2.pdf. Accessed: August 2026.
  4. Baudin E et al. Lung and thymic carcinoids: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2021;32[11]:1453-1455.
  5. McClellan K et al. Therapy Resistant Gastroenteropancreatic Neuroendocrine Tumors. Cancers. 2022;14[19]:4769.
  6. National Cancer Institute. Neuroendocrine tumor (NET). Available at: https://www.cancer.gov/pediatric-adult-rare-tumor/rare-tumors/rare-endocrine-tumor/carcinoid-tumor. Accessed: August 2026.
  7. Pavel M et al. Gastroenteropancreatic neuroendocrine neoplasms: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2020;31[7]:844-860.
  8. Neuroendocrine tumors. https://my.clevelandclinic.org/health/diseases/22006-neuroendocrine-tumors-net. Accessed: August 2026.
  9. Zekri J et al. Neuroendocrine tumor of the kidney. Diagnostic challenge and successful therapy. Urol Ann. 2019;11[4]:435-438.
  10. Singh et al. Patient-Reported Burden of a Neuroendocrine Tumor (NET) Diagnosis: Results From the First Global Survey of Patients With NETs. J Glob Oncol. 2017;3[1]:43–53.
  11. Survival Rates for Gastrointestinal Carcinoid Tumors. ACS website. Available at: https://www.cancer.org/cancer/types/gastrointestinal-carcinoid-tumor/detection-diagnosis-staging/survival-rates.html. Accessed: August 2026.
  12. Survival Rates for Lung Carcinoid Tumors. ACS website. Available at: https://www.cancer.org/cancer/types/lung-carcinoid-tumor/detection-diagnosis-staging/survival-rates.html. Accessed: August 2026.
  13. Durma AD et al. Epidemiology of Neuroendocrine Neoplasms and Results of Their Treatment with [177Lu]Lu-DOTA-TATE or [177Lu]Lu-DOTA-TATE and [90Y]Y-DOTA-TATE—A Six-Year Experience in High-Reference Polish Neuroendocrine Neoplasm Center. Cancers. 2023;15[22]:5466.
  14. Globocan: International Agency for Research on Cancer – Cancer Today. Available at: https://gco.iarc.who.int/media/globocan/factsheets/populations/900-world-fact-sheet.pdf. Accessed: August 2026.

CBZ-UK-001318

August 2026