European Commission decision expected Q3 2025

Ipsen that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion for Cabometyx (cabozantinib) for adult patients with unresectable or metastatic, well differentiated extra-pancreatic (epNET) and pancreatic (pNET) neuroendocrine tumors who have progressed following at least one prior systemic therapy other than somatostatin analogues. This recommendation is based on results from the CABINET Phase III trial presented at the European Society of Medical Oncology (ESMO) Congress 2024 and published in the New England Journal of Medicine.3,4 A final decision on the approval in the European Union is expected in the coming months.

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The number of people newly diagnosed with neuroendocrine tumors (NETs) is believed to be increasing, with a higher estimated prevalence than pancreatic or bladder cancer.5,6,7 Most forms of NETs develop slowly and can originate in various parts of the body,8 often requiring multiple lines of therapy as the disease progresses.1,2 Treatment options upon progression are often limited depending on primary tumor site and other factors, making it challenging to define optimal sequencing of treatments specific to individual patient needs.1,2,9 In particular, for the 27% of people diagnosed with lung NETs,10 there are no approved treatment options available upon progression on a non-somatostatin analogue-based systemic therapy.1,2

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“The significant efficacy data demonstrated in the CABINET Phase III trial have provided the opportunity to reframe conversations on care approaches for people living with advanced pancreatic and extra-pancreatic neuroendocrine tumors,” said Christelle Huguet, PhD, EVP and Head of Research and Development, Ipsen. “Today’s positive CHMP opinion confirms the potential to translate these data into meaningful benefits for patients and we look forward to receiving the final decision from the European Commission.”

The five-year survival rate is highly dependent on the primary site of disease. For advanced gastrointestinal and lung NETs, where the cancer has spread to distant parts of the body, the five-year survival rates are 68% and 55%, respectively.11,12 For people diagnosed with advanced pNET, however, the prognosis is poor, with a five-year survival rate of 23%.13

The positive CHMP opinion is 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 other than somatostatin analogues.3,4

  • 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).3,4
  • 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).3,4
  • Overall survival data were not mature at the time of the analyses and potentially confounded by the crossover design of the CABINET trial.3,4
  • 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.

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Source – GlobeNewswire