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

Most forms of neuroendocrine tumours (NETs) develop slowly, can originate in various parts of the body5 and require multiple lines of therapy as the disease progresses. Treatment options upon progression are often limited depending on primary tumour site and other factors, making it challenging to define optimal sequencing of treatments specific to individual patient needs. In particular, for the 27% of people diagnosed with lung NETs, there have been no approved treatment options upon progression on a prior therapy. 

“The complex nature of neuroendocrine tumours and lack of innovation in recent years has resulted in significant physical and emotional strain for patients as their disease progresses,” said Sandra Silvestri, MD, PhD, EVP and Chief Medical Officer, Ipsen. “We are pleased that for the first time, this approval offers a unique, simplified and efficacious treatment option upon progression, where few to no other options currently exist. We look forward to working with local health authorities to make cabozantinib available to even more patients, reinforcing our longstanding commitment to delivering transformational therapies in oncology.” 

NETs can have a significant impact on both individuals’ lives and wider society, with 71% of people reporting NETs having a negative effect on their daily life, with 92% reporting that they have to make lifestyle changes to accommodate their disease.  

“Sequential use of systemic therapies remains challenging in the different types of neuroendocrine tumour (NET) that may arise from various organ sites. The number of available therapies is limited and not all NET patients may benefit from currently approved therapies,” said Professor Marianne Pavel, Endocrinologist and NET expert at the Department of Medicine, Friedrich-Alexander University of Erlangen, Germany. “Advancements like those achieved through the CABINET Phase III study, as recognised by the approval for Cabometyx in a wide range of NET, is offering new treatment opportunities to delay disease progression in patients with well differentiated NET irrespective of the type of neuroendocrine tumour.” 

The EC approval was based on data from the CABINET Phase III trial which investigated Cabometyx versus placebo in people living with advanced pNETs or epNETs whose disease had progressed after prior systemic therapy. Final results from the trial, as presented at the 2024 European Society for Medical Oncology Congress and simultaneously published in the New England Journal of Medicine, demonstrated progression-free survival (PFS) benefits in favour of Cabometyx versus placebo:

• In the pNET cohort, at a median follow-up of 13.8 months, median PFS was 13.8 months for Cabometyx versus 4.4 months for placebo (hazard ratio (HR) 0.23 [95% confidence interval (CI) 0.12-0.42] p<0.001).
• In the epNET cohort, at a median follow-up of 10.2 months, median PFS based on local radiology review was 8.4 months for Cabometyx versus 3.9 months for placebo (HR 0.38 [95% CI 0.25-0.59] p<0.001). 
• Overall survival data were not mature at the time of the analyses and potentially confounded by the crossover design of the CABINET trial.
• The safety profile of Cabometyx observed in each cohort was consistent with its known safety profile; no new safety signals were identified.3,4 
• Per presentation at the Annual Society of Clinical Oncology Annual Meeting 2025, health-related quality of life was also found to be maintained or improved versus placebo.

Source: Ipsen