Cabozantinib’s EU Approval for Neuroendocrine Tumours

Neuroendocrine tumours (NETs) are a heterogeneous group of cancers that can arise in the intestine, pancreas, lungs and other organs. Because their behaviour varies widely, precise diagnosis—including histological classification and advanced imaging such as gallium PET—is essential before planning treatment.

Until recently, patients with advanced, progressive NETs after initial therapy had limited systemic options. That picture has changed with the European Union approval of cabozantinib, a drug developed by Ipsen. It becomes the first and only systemic therapy authorised for both pancreatic and extra-pancreatic well-differentiated NETs that are unresectable or metastatic and have progressed after at least one prior systemic therapy (other than somatostatin analogues).

The approval rests on the phase III CABINET study, which enrolled patients with pancreatic and extra-pancreatic NETs. Results showed that cabozantinib cut the risk of disease progression or death by 77% in pancreatic NETs and by 62% in extra-pancreatic NETs compared with placebo. Francesco Panzuto, president of the Italian NET association Itanet, called the data “very significant” given the limited options when disease advances.

Panzuto also highlighted the importance of national and European reference networks—Itanet and ENETS—in accelerating diagnosis (from a historical delay of four‑five years to around six months today) and ensuring patients reach centres where the latest therapies, including radioligands and now cabozantinib, are available.

Advertisement

What the New Therapy Means for the NET Treatment Landscape

Ipsen’s Commercial Prospects

With EU marketing authorisation, Ipsen gains a differentiated position in an underserved market. Cabozantinib is the first systemic therapy approved for both pancreatic and extra-pancreatic advanced NETs after prior therapy. The broad label could open a meaningful revenue stream, though the patient population is niche and competition from established somatostatin analogues and emerging radioligand treatments will limit the addressable pool to later lines.

Impact on Treatment Sequencing

The availability of cabozantinib adds a new layer to an already complex treatment algorithm. For patients whose tumours express somatostatin receptors and grow slowly, first-line analogues remain the standard. Radionuclide therapy has also gained ground. Cabozantinib enters at a later stage, potentially displacing other tyrosine kinase inhibitors or chemotherapies. Its strong efficacy data, particularly the 77% risk reduction in pancreatic NETs, may push it ahead of alternatives in that setting, though real-world sequencing will depend on individual tumour biology, tolerability and local guidelines.

The Role of Reference Networks

The story underscores the value of coordinated clinical networks. Itanet’s work, mirrored by ENETS at European level, has sharply reduced diagnostic delays and funneled patients to centres equipped with advanced diagnostics and therapies. With cabozantinib being a specialist-prescribed drug, such networks become even more critical for equitable access and appropriate patient selection. The Italian experience shows that structured referral pathways can translate clinical advances into faster, better‑informed care.

Navigating the New Option: Advice for Patients and Centres

For patients:

Advertisement
  • If you have a diagnosis of advanced, progressive neuroendocrine tumour of the pancreas or other site and have already received systemic therapy, ask your oncologist whether cabozantinib is a suitable next step. The drug is available in the EU, though local reimbursement may vary.
  • Seek care at a specialised NET centre. In Italy, centres affiliated with Itanet can provide access to the latest therapies and clinical trials. Similar networks exist across Europe through ENETS.

For clinicians and healthcare institutions:

  • Update internal guidelines to incorporate cabozantinib as a post-progression option, taking into account the primary tumour site and prior lines of therapy.
  • Strengthen referral links between general hospitals and NET reference centres to ensure patients are assessed for novel treatments without unnecessary delay.

Risk & Opportunity Assessment

Commercial RiskLowThe drug is already approved and commercialised. Pricing and reimbursement negotiations are the main source of short‑term uncertainty, but the need for effective later‑line therapy supports adoption.
Competitive RiskMediumSomatostatin analogues and radionuclide therapy dominate earlier lines. Cabozantinib may compete directly with other TKIs or off‑label chemotherapies in the post‑progression setting, but its unique broad-label position offers a competitive moat.
Regulatory RiskLowThe EMA has granted full approval based on robust phase III data. No major safety signals were highlighted in the CABINET study.
Reputation RiskLowThe therapy is endorsed by a leading NET association president; no controversies or adverse publicity are attached.
Technology DisruptionMediumAs a multi‑kinase inhibitor, cabozantinib introduces a complementary mechanism of action. It does not replace radioligand therapy but adds a new option for treatment‑resistant patients, making the overall NET armamentarium more diversified.
Commercial OpportunityHighFirst‑to‑market status for both pancreatic and extra‑pancreatic advanced NETs in the EU gives Ipsen a near‑term monopoly window in an area of high unmet need. Even with a modest patient population, the premium pricing typical of targeted oncology drugs can generate significant revenue.