National Institute for Health and Care Excellence (NICE) recommends cabozantinib as an option for treating advanced hepatocellular carcinoma (HCC), a type of primary liver cancer, in adults who have had sorafenib, only if they have Child-Pugh grade A liver impairment and an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.

The recommendation is based on the results of the global placebo-controlled CELESTIAL phase 3 pivotal trial which included a patient population receiving cabozantinib in second or third line after treatment with sorafenib.

“People living with the most common form of primary liver cancer, HCC, have a poor prognosis and there are very few treatments available. Often, the disease is diagnosed so late that palliative care is the only option. The nurses on the British Liver Trust’s helpline hear every day from patients who are completely devastated.

They live with uncertainty, hopelessness and often stigma and isolation due to the image of liver cancer. Treatments that buy extra time can not only positively impact those individuals but can also have a huge positive impact on families and the wider community, so today’s recommendation is an important step forward in the treatment of HCC.” said Vanessa Hebditch, Director of Communications & Policy, British Liver Trust

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 8.0 months with placebo (HR 0.76, 95 percent CI 0.63-0.92; p=0.005).

Corresponding to this survival benefit, a longer duration of progression-free survival was also observed: the median progression-free survival (PFS) was 5.2 months with cabozantinib and 1.9 months with placebo (HR 0.44, 95 percent CI 0.36-0.52; p<0.0001).

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.

Professor Tim Meyer, Professor of Experimental Cancer Medicine at UCL Cancer Institute and Honorary Consultant in Medical Oncology at the Royal Free Hospital added, “HCC is the third leading cause of cancer death worldwide and has one of the lowest five-year survivals of all cancers. For patients with advanced disease, treatment options remain limited, and outcomes are poor.

Today’s recommendation by NICE will make cabozantinib available to such patients and provides an important addition to the therapeutic landscape for HCC. Cabozantinib has been proven to improve survival, slow disease progression and delay deterioration in disease-related symptoms when used as second- or third-line treatment, and the decision to allow its use will be welcomed by doctors treating this challenging disease.”

Manjinder Bains, Medical Director, Ipsen UK and Ireland comments, “We are pleased with the decision to make cabozantinib available to eligible liver cancer patients in England and Wales.

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. 

Source: Ipsen