Ipsen has received approval from the European Commission (EC) for its Exelixis-partnered Cabometyx (cabozantinib) in advanced neuroendocrine tumours (NETs).
The drug has been authorised to treat unresectable or metastatic, well-differentiated pancreatic and extra-pancreatic NETs in adults who have progressed following at least one prior systemic therapy other than somatostatin analogues.
Also called neuroendocrine neoplasms, NETs are tumours that arise from the neuroendocrine cells found throughout the body. Most forms develop slowly over a number of years and require multiple lines of therapy as the disease progresses; however, treatment options following progression are often limited depending on the primary tumour site and other factors.
Cabometyx is now the first systemic therapy to be approved in the EU for previously treated unresectable or metastatic NETs, regardless of tumour site, grade or previous non-somatostatin analogue-based systemic therapy.
The drug is designed to inhibit multiple receptor tyrosine kinases, reducing the growth and spread of the disease, and is already approved in the EU to treat certain cases of renal cell carcinoma, thyroid carcinoma and hepatocellular carcinoma.
The EC’s decision follows a recent recommendation from the European Medicines Agency’s human medicines committee and was supported by results from the late-stage CABINET trial, in which Cabometyx demonstrated continued improvement in the primary endpoint of progression-free survival (PFS).
Among patients with pancreatic NETs, median PFS was 13.8 months for Cabometyx versus 4.4 months for placebo at a median follow-up of 13.8 months. For those with extra-pancreatic NETs, median PFS was 8.4 months and 3.9 months for Cabometyx and placebo, respectively, at a median follow-up of 10.2 months.
Sandra Silvestri, executive vice president and chief medical officer at Ipsen, said: “The complex nature of NETs 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.”
Exelixis holds exclusive rights to develop and commercialise Cabometyx in the US, and has granted Ipsen exclusive rights to the drug outside of the US and Japan, and exclusive rights to Takeda in Japan.