French biopharmaceutical company Ipsen has received European Commission (EC) approval for Cabometyx (cabozantinib) to treat neuroendocrine tumours.
Cabometyx functions as a small-molecule inhibitor targeting multiple receptor tyrosine kinases, including VEGFRs, MET, RET, and the TAM family (TYRO3, MER, AXL).
The kinases play roles in oncogenesis, metastasis, tumour angiogenesis, drug resistance, immune modulation, and tumour microenvironment maintenance.
Cabometyx is indicated for adult patients with unresectable or metastatic, well-differentiated pancreatic (pNET) and extra-pancreatic (epNET) neuroendocrine tumours.
The EC approval applies to those patients who have progressed after at least one prior systemic therapy, excluding somatostatin analogues.
Cabometyx was originally developed by Exelixis, a biopharmaceutical company focused on oncology. Ipsen secured exclusive rights to the drug outside the US and Japan.
In Japan, Takeda Pharmaceutical Company holds exclusive rights for future indications, while Exelixis retains rights within the US.
Ipsen EVP and chief medical officer Sandra Silvestri said: “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.
“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 Cabometyx available to even more patients, reinforcing our longstanding commitment to delivering transformational therapies in oncology.”
The EC approval is based on data from the Phase 3 CABINET trial, which compared Cabometyx to a placebo in patients with advanced pancreatic or epNET.
The trial included 298 patients in the US and was sponsored by the National Cancer Institute, part of the National Institutes of Health.
It showed that Cabometyx improved progression-free survival (PFS) compared to placebo.
In the pancreatic neuroendocrine tumour cohort, the median PFS was 13.8 months for Cabometyx versus 4.4 months for placebo.
In the extra-pancreatic cohort, the median PFS was 8.4 months for Cabometyx compared to 3.9 months for placebo.
Overall survival data were not mature due to the crossover design of the CABINET trial.
The safety profile of Cabometyx was consistent with previous findings, with no new safety signals identified.
In addition, health-related quality of life was maintained or improved compared to placebo.
