Ipsen’s cabozantinib receives NICE approval for advanced hepatocellular carcinoma

Ipsen’s cabozantinib has been recommended by the National Institute for Health and Care Excellence (NICE) as an option for patients with previously treated advanced hepatocellular carcinoma (HCC).

Specifically, the recommendation is for adult patients who have had sorafenib – the standard initial treatment for advanced disease – only if they have Child-Pugh grade A liver impairment and an Eastern Cooperative Oncology Group performance status of zero or one.

NICE’s decision is based on positive results from the global placebo-controlled CELESTIAL phase 3 pivotal trial in patients in the second or third line after treatment with sorafenib.

The trial met its primary endpoint of overall survival (OS), with cabozantinib providing a statistically significant improvement in OS compared with placebo in patients with advanced HCC who have been previously treated with sorafenib.

The median OS in the overall population was 10.2 months with cabozantinib and eight months with placebo. A longer duration of progression-free survival (PFS) was also observed, with a PFS of 5.2 months in cabozantinib-treated patients and 1.9 months in those receiving placebo.

In terms of safety, adverse events were consistent with the known safety profile of cabozantinib, and the rate of high-grade adverse events in the cabozantinib group was approximately twice that observed in the placebo group.

Cabozantinib is an oral tyrosine kinase inhibitor administered daily with routine outpatient monitoring. Specifically, cabozantinib inhibits tyrosine kinases, including vascular endothelial growth factor receptors 1, 2, and 3, MET, and AXL, which are implicated in the progression of HCC and the development of resistance to sorafenib.

HCC is the most common type of primary liver cancer in the UK. Despite improvements in screening, surveillance rules, and imaging, most patients are diagnosed in the advanced stages of the disease, when cirrhosis is present and surgery is rarely an option, with the disease considered incurable.

Without treatment, the median survival of advanced HCC ranges between four and eight months, the company reported, and current second and third line treatment options for those living with HCC are limited.

Manjinder Bains, medical director, Ipsen UK and Ireland, said: “At this stage of the disease, it is critical to intervene with another treatment that can prolong survival and delay progression, which we know can make such a difference to patients and their families living with this devastating condition.”