By Fineline Cube 
France-based pharmaceutical company Ipsen S.A. (EPA: IPN, OTCMKTS: IPSEY) announced that its anticancer drug Cabometyx (cabozantinib) has been approved by the European Commission for the treatment of adult patients with unresectable or metastatic pancreatic and extra-pancreatic neuroendocrine tumors (pNET and epNET). This approval is for patients who have experienced disease progression after at least one prior systemic therapy that does not include somatostatin analogues. This marks the first EU approval of a systemic treatment for this patient population.
Drug Mechanism and Previous Approvals
Cabometyx is an oral small-molecule tyrosine kinase inhibitor targeting multiple key pathways, including VEGFR, MET, and RET. It has already been approved in multiple countries for treating renal cell carcinoma, hepatocellular carcinoma, and thyroid cancer.
Clinical Trial Results
The approval is based on results from the CABINET Phase III clinical trial. In the pNET population, Cabometyx reduced the risk of disease progression or death by 77% compared to placebo, extending median progression-free survival (PFS) from 4.4 months to 13.8 months (HR=0.23, p<0.001). In the epNET population, it reduced the risk by 62%, with median PFS extended from 3.9 months to 8.4 months (HR=0.38, p<0.001). Overall survival data remain immature. The safety profile of Cabometyx was consistent with previous findings, with no new safety signals observed.
Significance for Patients
Treatment options for patients with progressive neuroendocrine tumors have been limited, especially for those with lung-origin NETs who have long lacked approved therapies. This approval provides a new treatment option for this challenging clinical setting and significantly broadens the indications for Cabometyx. It represents an important advancement in the treatment of neuroendocrine tumors.-Fineline Info & Tech