CABOMETYX® (cabozantinib) for pNET and epNET

18th October 2025. Exelixis Announces Results from Subgroup Analysis of CABINET Phase 3 Pivotal Trial Evaluating CABOMETYX® (cabozantinib) in Advanced Lung and Thymic Neuroendocrine Tumors at ESMO 2025 | Exelixis, Inc.

– CABOMETYX reduced the risk of disease progression or death by 81% versus placebo in patients with advanced lung or thymic neuroendocrine tumors (NET) –

– The lungs are the second most common NET site of origin, yet limited treatment options are available

Read announcement from Exelixis here.

23rd September 2025. UK MHRA grants marketing authorization

Ipsen (Euronext: IPN) announced today that UK MHRA has granted marketing authorization for cabozantinib in adult patients with unresectable or metastatic, well differentiated pNETs and epNETs who have progressed following at least one prior systemic therapy other than SSAs.

This does not mean the drug is available right now.

“This U.K. marketing authorisation marks a critical milestone in making a licensed and efficacious treatment option available to patients upon progression. We are actively engaging with the National Institute for Health and Care Excellence (NICE) to ensure that individuals living with this often overlooked form of cancer in the U.K. have timely access to this approved therapeutic option,” said Dr Ian Gray, U.K. & Ireland Interim Medical Director at Ipsen.

Cabozantinib has been submitted for review by NICE, with a decision expected later in 2025 but is now late (as at 7th Jan 2026). I will keep you updated as usual.

Read announcement from Ipsen here

24th July 2025. What about EU/UK and elsewhere

PARIS, FRANCE, 24 July 2025 – Ipsen announced today that the European Commission has approved Cabometyx^® (cabozantinib) for adult patients with unresectable or metastatic, well differentiated pancreatic (pNET) and extra-pancreatic (epNET) neuroendocrine tumors who have progressed following at least one prior systemic therapy other than somatostatin analogues.

Cabometyx® is the first and only systemic therapy approved in the European Union for previously treated neuroendocrine tumours, regardless of tumour site, grade or previous non-somatostatin analogue-based systemic therapy

In the meantime, I would advise anyone not in USA, to contact their doctors asking about any potential access prior to national approvals. I have anecdotal evidence this is already happening in the Netherlands.

Read announcement from Ipsen here.

21 May 2025 – Dr Thor explains NETs and in particular the Phase 3 CABINET Trial and subsequent approval

Click here to see a series of short videos. Fascinating conversation.

ESMO24: Jennifer Chan, MD, MPH CABINET Trial | Dana-Farber Cancer Institute

Jennifer Chan, MD, MPH, highlights the significance of the FDA approval of cabozantinib for the treatment of patients with pNET and epNET.

26 March 2025. US FDA approves for pNET and epNET

FDA approves cabozantinib for adults and paediatric patients 12 years of age and older with pNET and epNET

On March 26, 2025, the Food and Drug Administration approved cabozantinib (Cabometyx, Exelixis, Inc.) for adult and paediatric patients 12 years of age and older with previously treated, unresectable, locally advanced or metastatic, well-differentiated pancreatic neuroendocrine tumours (pNET) and well-differentiated extra-pancreatic neuroendocrine tumours (epNET).

What is this drug prescribed for?

Full prescribing information for Cabometyx (Cabozantinib) for pNET and epNET is click here.

What do I need to know including insurance and financial support? (US patients)

The patient handbook produced by Exelixis can be found by clicking here – this should answer queries including financial support and insurance.

Please note the use of the term ‘Extrapancreatic Neuroendocrine Tumours’. It simply means something that originates outside of the pancreas.

Another tool?

Yes. When you update yourself below and note the wording from the manufacturer above, it becomes evident that Cabozantinib is probably not a first line treatment. Rather it is a tool to be used downstream after current treatments still result in progression. The trial was based on those with advanced Neuroendocrine Tumours. During that trial, cabozantinib, as compared with placebo, was used on those that had progressed after previous therapy with Lu-177 dotatate or targeted agents, including everolimus or sunitinib. The output data looks like it is a great option for those in that position and offers hope.

Please note the use of the term ‘Extrapancreatic Neuroendocrine Tumours’. It simply means something that originates outside of the pancreas.

Announcement of final data from the Phase 3 trial

There has been a tremendous amount of postings about this drug since the phase 3 trial sponsors produced their initial data. Since then there has been a Supplemental New Drug Application (sNDA) for Cabozantinib for Patients with Advanced Neuroendocrine Tumours. The FDA assigned a Prescription Drug User Fee Act target action date of April 3, 2025 (i.e. a new or amended approval will be decided by then). The sNDA Application is based on results from the phase 3 CABINET pivotal trial, in which cabozantinib provided a statistically significant and clinically meaningful improvement in progression-free survival versus placebo. Cabozantinib, as compared with placebo, significantly improved progression-free survival in patients with previously treated, progressive advanced extrapancreatic or pancreatic neuroendocrine tumours. Adverse events were consistent with the known safety profile of cabozantinib. (Funded by the National Cancer Institute and others; CABINET ClinicalTrials.gov number, N CT03375320.) An excellent summary of the final data can be found on the manufacturers website – See Reference 2 below. For those with access to The New England Journal of Medicine, you may be able to access the full report here.

You may be wondering why Ipsen is involved? I also discovered that there is an exclusive licensing Agreement with the manufacturers (Exelixis) and Ipsen (of Lanreotide fame) to commercialize and develop Cabozantinib in regions outside the United States and Japan Also, for Europeans, Ipsen has submitted an extension of the Marketing Authorization to the European Medicines Agency (EMA) which means the drug could potentially be approved in Europe with a similar timescale to the US FDA sNDA above. Lets hope so.

What is Cabozantinib?

Cabozantinib is an oral drug which works by blocking the growth of new blood vessels that feed a tumour. In addition to blocking the formation of new blood cells in tumours, Cabozantinib also blocks pathways that may be responsible for allowing cancers cells to become resistant to other “anti-angiogenic” drugs. It is a type of drug called a growth blocker. Cabozantinib has been studied or is already in research studies as a possible treatment for various types of cancer, including prostate cancer, ovarian cancer, brain cancer, thyroid cancer, lung cancer, and kidney cancer. Also known by the brand name of Cabometyx®

Growth blockers are a type of targeted therapy and include tyrosine kinase inhibitors, proteasome inhibitors, mTOR inhibitors, PI3K inhibitors, histone deacetylase inhibitors and hedgehog pathway blockers. Cabozantinib is a tyrosine kinase inhibitor (TKI). They block chemical messengers (enzymes) called tyrosine kinases. Tyrosine kinases help to send growth signals in cells so blocking them stop the cell growing and dividing. Some TKIs can block more than one tyrosine kinase and these are known as multi-TKIs.

cabozantinib-picture — Example action of Cabozantinib

So Cabozantinib is a targeted therapy and growth blocker just like Everolimus (Afinitor) and Sunitinib (Sutent). Very technical process but in the simplest of terms, Cabozantinib is designed to disrupt the actions of VEGF (a growth factor) and MET (a growth factor receptor) which promote spread of cancerous cells through the growth of new blood vessels. Whilst we are on this subject, please note Everolimus (Afinitor) is an mTOR inhibitor and Sunitinib (Sutent) is a tyrosine kinase inhibitor. Many people think these drugs are a type of chemo – that is incorrect, these are targeted therapies. See more on this by clicking here.

During my research, I found that it is an existing drug approval for other cancers and even has a connection to a type of Neuroendocrine Cancer called Medullary Thyroid Cancer (MTC). Cabozantinib, under the brand name of ‘Cometriq’ was approved by the FDA and other national approval agencies in 2012 for use in MTC which is frequently associated with Multiple Endocrine Neoplasia 2 (MEN2). Read more about Cometriq here. It’s also been approved by the FDA for advanced renal cell carcinoma (RCC) (branded as Cabometyx).

The history of the clinical trial covering most NETs can be found below.

Phase 3 CABINET Clinical Trial – Cabozantinib (Cabometyx) for Advanced Neuroendocrine Tumours

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