Ipsen  announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending the approval of Cabometyx® (cabozantinib) for the treatment of adult patients with unresectable or metastatic, well-differentiated pancreatic (pNET) and extra-pancreatic (epNET) neuroendocrine tumors (NETs) who have progressed following at least one prior systemic therapy other than somatostatin analogues.

The European Commission’s final decision is expected in the third quarter of 2025.

This recommendation is based on the pivotal CABINET Phase III trial, the results of which were presented at the ESMO Congress 2024 and published in the New England Journal of Medicine. The trial demonstrated that Cabometyx significantly reduced the risk of disease progression or death compared with placebo, offering a meaningful advancement for patients with limited treatment options.

“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 at 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.”

CABINET Phase III Trial Highlights

• pNET Cohort:

  • Median progression-free survival (PFS): 13.8 months with Cabometyx vs 4.4 months with placebo

  • Hazard ratio (HR): 0.23 [95% CI: 0.12–0.42]; p < 0.001

• epNET Cohort:

  • Median PFS: 8.4 months with Cabometyx vs 3.9 months with placebo

  • HR: 0.38 [95% CI: 0.25–0.59]; p < 0.001

• The safety profile was consistent with existing data for Cabometyx, and no new safety signals were observed.

• Health-related quality of life was maintained or improved in treated patients.¹⁴

A Significant Milestone in the Management of NETs

Neuroendocrine tumors, which can originate in various organs such as the pancreas, gastrointestinal tract, or lungs, are on the rise globally and often require complex, multi-line treatment strategies. Importantly, 27% of NETs occur in the lungs, a group for which no approved systemic therapies exist after progression on non-somatostatin analogue-based treatment.¹˒² If approved, Cabometyx would address this urgent unmet need.

The five-year survival rate for advanced NETs varies widely, from 68% in gastrointestinal NETs to 23% in advanced pNETs, highlighting the need for effective second-line treatments.¹¹˒¹²˒¹³