Research & Development
AstraZeneca announces grant of Orphan Drug Designation in the US to Imfinzi and tremelimumab for liver cancer treatment
21 January 2020 -

AstraZeneca Plc (STO:AZN) (LON:AZN) (NYSE:AZN), a global, science-led biopharmaceutical company, reported on Monday that Imfinzi (durvalumab) and tremelimumab, an anti-CTLA4 antibody and potential new medicine, have both been granted Orphan Drug Designation (ODD) in the US for the treatment of hepatocellular carcinoma (HCC), the most common type of liver cancer.

The US Food and Drug Administration (FDA) grants ODD to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.

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

Currently, Imfinzi is not approved to treat HCC in any country, alone or in combination with tremelimumab.

HIMALAYA is a randomised, open-label, multicentre, global phase III trial of Imfinzi monotherapy and the combination of Imfinzi 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. This 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.

Imfinzi is approved in the curative-intent setting of unresectable, stage III non-small cell lung cancer (NSCLC) after chemoradiation therapy in 54 countries, including the US, Japan, China and across the EU, based on the phase III PACIFIC trial. Imfinzi is also approved for previously treated patients with advanced bladder cancer in 11 countries, including the US.

In addition, Imfinzi is under Priority Review with FDA for the treatment of patients with previously untreated extensive-stage small cell lung cancer (SCLC) in combination with chemotherapy. A Prescription Drug User Fee Act date is set for the first quarter of 2020.

AstraZeneca is focussed on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in the three therapy areas of Oncology, Cardiovascular, Renal and Metabolism and Respiratory.

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