Ipsen’s response to NICE’s decision on Cabometyx® (cabozantinib) for previously treated differentiated thyroid cancer unsuitable for or refractory to radioactive iodine

SLOUGH, UK, 5th April 2023 – Ipsen is disappointed that the National Institute for Health and Care Excellence (NICE) has issued preliminary guidance which does not recommend Cabometyx® (cabozantinib) for previously treated differentiated thyroid cancer (DTC) unsuitable for or refractory to radioactive iodine and which has progressed after systemic treatment.[i]

There are 3,865 new thyroid cancer cases in the UK every year[ii] and DTC is the most common form, accounting for ~90-95% of all diagnosed cases.[iii],[iv] Females are much more likely to be diagnosed with thyroid cancer, making up 72% of the thyroid cancer cases in the UK2 and DTC tends to affect people of working age (the median age of diagnosis is between 45 and 49 years)2 . Whilst the outlook is good for most DTC patients, there are a few patients with advanced disease in whom radioiodine does not work, so called radioiodine refractory DTC (RAI-R DTC), who then require chemotherapy treatment.4,[v],[vi],[vii] However, if their disease progresses while on NICE recommended first-line chemotherapy treatment, there are currently no treatment options in England and Wales unless they have targetable genetic alterations (NTRK and RET fusions).[viii] For the majority of these patients who do not have genetic alterations the prognosis is very poor[ix],[x] and unlike patients in Scotland,[xi],[xii] they have been without a second-line treatment option recommendations since 2018.

Guy Oliver, General Manager, Ipsen UK and Ireland comments, “This preliminary decision by NICE is disappointing for all those living with DTC in England and Wales. Treatment options are extremely limited for people living with DTC and we believe cabozantinib addressed a significant unmet need in this patient population. The decision is also surprising due to the fact that cabozantinib fell within NICE’s cost-effectiveness range, especially as it was acknowledged by the NICE committee that there is an unmet need in England and Wales for an effective second-line treatment. It is yet another example of how our health systems in the UK are not fit for purpose and the need for reform is now critical to ensure innovative medicines for cancers are made available to those who have no other treatment options and a very poor prognosis. We will be responding to the NICE consultation and remain committed to working collaboratively with NICE and the NHS to achieve a positive outcome for people living with DTC.”

 NICE will proceed to a public consultation on the preliminary recommendation, in advance of the final recommendation which is expected later in 2023.




About cabozantinib

Cabozantinib inhibits tyrosine kinases, including potent inhibitor of mesenchymal epithelial transition (MET), growth arrest-specific protein 6 receptor (AXL), rearranged during transfection (RET) and vascular endothelial growth factor receptor 2 (VEGFR2), all of which are known to be important in the pathogenesis of thyroid cancer, specifically DTC.[xiii],[xiv],[xv],[xvi],[xvii]


About differentiated thyroid cancer (DTC)

There are 3,865 new thyroid cancer cases in the UK every year2 and DTC is most common form, accounting for ~90-95% of all diagnosed cases.3,4 Females are much more likely to be diagnosed with thyroid cancer, making up 72% of the thyroid cancer cases in the UK.2

Expected survival is markedly worse in patients who have progressed on first-line systemic treatment.9,10 Based on real-world data, the median survival for patients who have not received salvage therapy after progressing from a single agent TKI ranges from 10 to 22 months.9,10 There are currently no other active treatment options for people living with DTC after the first line systemic treatment has failed in England and Wales, and prognosis is very poor.9,10

For people whose condition has progressed on systemic therapy (lenvatinib or sorafenib), and who are no longer having treatment with them, the only remaining option is best supportive care. This typically comprises thyroid stimulating hormone (TSH) suppression and ongoing imaging, with palliative radiotherapy and symptom relief where necessary.1


About Ipsen 

Ipsen is a global, mid-sized biopharmaceutical company focused on transformative medicines in Oncology, Rare Disease and Neuroscience. With Specialty Care sales of €2.6bn in FY 2021, Ipsen sells medicines in over 100 countries. Alongside its external innovation strategy, the Company’s research and development efforts are focused on innovative and differentiated technological platforms located in the heart of leading biotechnological and life-science hubs: Paris-Saclay, France; Oxford, U.K.; Cambridge, U.S. Ipsen has around 5,700 colleagues worldwide and is listed in Paris (Euronext: IPN). In the UK Ipsen employs 750 people across three sites, Research & Development (Abingdon, Oxford), Pharmaceutical Development and Manufacturing (Wrexham, Wales) and Commercial headquarters (Slough, Berkshire). For more information, visit https://www.ipsen.com/uk-ireland/.



For further information:



Jessica Greenman

Communications and Patient Affairs Manager

UK & Ireland Global Hub


+44 (0)7731 981010




April 2023


[i] National Institute for Health and Care Excellence (NICE). Draft guidance consultation – Cabozantinib for previously treated advanced differentiated thyroid cancer unsuitable for or refractory to radioactive iodine. Issued 05 April 2023.

[ii] Cancer Research UK. Thyroid Cancer Incidience Statistics. Available at https://www.cancerresearchuk.org/health-professional/cancer-statistics/statistics-by-cancer-type/thyroid-cancer/incidence. Last accessed April 2023.

[iii] Kent WD, Hall SF, Isotalo PA, Houlden RL, George RL, Groome PA. Increased incidence of differentiated thyroid carcinoma and detection of subclinical disease. Canadian Medical Association Journal 2007;177:1357-61.

[iv] Tumino D, Frasca F, Newbold K. Updates on the management of advanced, metastatic, and radioiodine refractory differentiated thyroid cancer. Frontiers in Endocrinology 2017;8.

[v] Babu G & Kainickal CT. Update on the systemic management of radioactive iodine refractory differentiated thyroid cancer (Review). Mol Clin Oncol 2021. 14: 35.

[vi] Filetti S, Durante C, Hartl D, et al. Thyroid cancer: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Annals of Oncology 2019. 30: 1856–1883.

[vii] Schlumberger M, Tahara M, Wirth LJ, et al. Lenvatinib versus Placebo in Radioiodine-Refractory Thyroid Cancer. New England Journal of Medicine 2015. 372: 621–630.

[viii] National Institute for Health and Care Excellence. Lenvatinib and sorafenib for treating differentiated thyroid cancer after radioactive iodine. Published 08 August 2018. Available from: https://www.nice.org.uk/guidance/ta535. Last accessed April 2023.

[ix] Oh HS, Shin DY, Kim M, Park SY, Kim TH, Kim BH, et al. Extended real-world observation of patients treated with sorafenib for radioactive iodine-refractory differentiated thyroid carcinoma and impact of lenvatinib salvage treatment: A Korean multicenter study. Thyroid 2019;29:1804-10.

[x] Brilli L, Dalmiglio C, Pilli T, Barbato F, Maino F, Capezzone M, et al. Improvement of overall survival using TKIs as salvage therapy in advanced thyroid carcinoma: Real-Life data on a single center experience. Journal of Clinical Medicine 2021;10:384.

[xi] Lenvatinib (Lenvima). Scottish Medicines Consortium. Available at: https://www.scottishmedicines.org.uk/medicines-advice/lenvatinib-lenvima-fullsubmission-117916/. Last accessed April 2023.

[xii] Sorafenib (Nexavar). Scottish Medicines Consortium. Available at: https://www.scottishmedicines.org.uk/medicines-advice/sorafenib-nexavar-fullsubmission-105515/. Last accessed April 2023.

[xiii] Harshman LC, Choueiri TK. Targeting the hepatocyte growth factor/c-Met signaling pathway in renal cell carcinoma. Cancer J. 2013;19(4):316–23.

[xiv] Sennino B, Ishiguro-Oonuma T, Wei Y, Naylor RM, Williamson CW, Bhagwandin V, et al. Suppression of tumor invasion and metastasis by concurrent inhibition of c-Met and VEGF signaling in pancreatic neuroendocrine tumors. Cancer Discov. 2012;2(3):270–87.

[xv] Yakes FM, Chen J, Tan J, Yamaguchi K, Shi Y, Yu P, et al. Cabozantinib (XL184), a novel MET and VEGFR2 inhibitor, simultaneously suppresses metastasis, angiogenesis, and tumor growth. Mol Cancer Ther. 2011;10(12):2298–308.

[xvi] Rankin EB, Fuh KC, Castellini L, Viswanathan K, Finger EC, Diep AN, et al. Direct regulation of GAS6/AXL signaling by HIF promotes renal metastasis through SRC and MET. Proc Natl Acad Sci U S A. 2014 Sep 16;111(37):13373–8.

[xvii] Gibney GT, Aziz SA, Camp RL, Conrad P, Schwartz BE, Chen CR, et al. c-Met is a prognostic marker and potential therapeutic target in clear cell renal cell carcinoma. Ann Oncol Off J Eur Soc Med Oncol. 2013 Feb;24(2):343–9.

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