AstraZeneca has secured orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for Imfinzi (durvalumab) and tremelimumab to treat liver cancer called hepatocellular carcinoma (HCC).
Imfinzi is a human monoclonal antibody that binds to PD-L1 and restricts the interaction of PD-L1 with PD-1 and CD80, helping to oppose the tumour’s immune-evading tactics and release the inhibition of immune responses.
Tremelimumab, a human monoclonal antibody, is a potential new medicine that can target the activity of cytotoxic T-lymphocyte-associated protein 4 (CTLA-4). It is being assessed in a clinical trial programme in combination with Imfinzi in NSCLC, SCLC, bladder cancer, head and neck cancer and liver cancer.
The phase III HIMALAYA study is evaluating Imfinzi and the combination of Imfinzi plus tremelimumab in patients with unresectable, advanced HCC who were not treated with prior systemic therapy, as well as not eligible for locoregional therapy.
HIMALAYA is claimed to be the first study to evaluate dual immune checkpoint blockade in the first-line advanced HCC setting.
HIMALAYA is a randomised, open-label, multicentre and global phase III trial designed to evaluate Imfinzi as monotherapy and in combination tremelimumab against the standard-of-care medicine sorafenib in patients with unresectable, advanced HCC who have not been treated with prior systemic therapy and are not eligible for locoregional therapy. Sorafenib is a multi-kinase inhibitor.
According to AstraZeneca, the primary endpoint is overall survival and major secondary endpoints comprise of objective response rate and progression-free survival.
Imfinzi has not yet secured approval for the treatment of HCC in any country, alone or in combination with tremelimumab.
AstraZeneca oncology R&D executive vice president José Baselga said: “Many patients with liver cancer are diagnosed and treated only after the disease is advanced, and there is an urgent need for new effective and tolerable treatments.
“We are eager to bring new potential options to these patients and look forward to the results of our ongoing Phase III HIMALAYA trial later this year.”
In November 2019, AstraZeneca secured priority review from the FDA for Imfinzi to treat extensive-stage small cell lung cancer (SCLC) in previously untreated patients.