Durvalumab & tremelimumab get orphan drug desig for HCC in US

March 6, 2020 0 By FM

Durvalumab and tremelimumab have both been granted Orphan Drug Designation (ODD) in the US for the treatment of hepatocellular carcinoma (HCC).

The phase III HIMALAYA trial is testing durvalumab and the combination of durvalumab plus tremelimumab in patients with unresectable, advanced HCC who have not been treated with prior systemic therapy and are not eligible for locoregional therapy. HIMALAYA is the first trial to test dual immune checkpoint blockade in the 1st-line advanced HCC setting.

HIMALAYA is a randomised, open-label, multicentre, global phase III trial of durvalumab monotherapy and the combination of durvalumab and tremelimumab vs. the standard-of-care medicine sorafenib, a multi-kinase inhibitor, in patients with unresectable, advanced HCC who have not been treated with prior systemic therapy and are not eligible for locoregional therapy. The trial is being conducted in 189 centres across 16 countries including in the US, Canada, Europe, South America and Asia. The primary endpoint is overall survival and key secondary endpoints include objective response rate and progression-free survival.

Durvalumab is a human monoclonal antibody that binds to PD-L1 and blocks the interaction of PD-L1 with PD-1 and CD80. 

Tremelimumab targets the activity of cytotoxic T-lymphocyte-associated protein 4 (CTLA-4).