Jan 20,2020: AstraZeneca’s Imfinzi (durvalumab) and tremelimumab, an anti-CTLA4 antibody and potential new medicine,both have been granted Orphan Drug Designation (ODD) in US for the treatment of hepatocellular carcinoma (HCC), the most common type of the liver cancer.
The U.S. FDA grants ODD to medicines proposed for the treatment, diagnosis or prevention of rare diseases or disorders that affect less than 200,000 people in the US. Liver cancer is the third leading cause of cancer death worldwide for the patients with unresectable or advanced disease, only 13% are living five years after diagnosis.
The Phase III HIMALAYA trial is testing Imfinzi and the combination of Imfinzi plus tremelimumab in the patients with unresectable, advanced HCC who have not been treated with previous systemic therapy and are not suitable for locoregional therapy (treatment localised to the liver).
HIMALAYA is the first trial in order to test dual immune checkpoint blockade in the 1st-line advanced HCC setting. At present Imfinzi is not approved in order to treat HCC in any country, alone or in combination with tremelimumab. https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2020/imfinzi-and-tremelimumab-granted-orphan-drug-designation-in-the-us-for-liver-cancer-20012020.html