AstraZeneca’s PD-L1 Imfinzi, and anti-CTLA4 antibody tremelimumab, have been granted orphan drug designations (ODD) in the US for liver cancer.
The FDA has granted the orphan drug status on the basis of early data from AZ’s Study 22, an exploratory phase 1/2 trial testing Imfinzi (durvalumab) in patients with advanced hepatocellular carcinoma (HCC).
AZ is also testing Imfinzi and Imfinzi plus tremelimumab in the phase 3 HIMALAYA trial, as a first-line treatment for unresectable, advanced HCC, for which data is expected in the second half of this year.
HCC is the most common form of liver cancer, with more than 750,000 people worldwide diagnosed with the disease every year. Out of that number of new cases, 42,000 people were diagnosed with HCC in the US last year.
The FDA grants an ODD to drugs intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the US.
According to AZ, HIMALAYA is the first trial to test dual immune checkpoint blockade in the first-line advanced HCC setting. The detailed results from the ongoing trial are expected later this year.
“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,” said José Baselga, executive vic president, oncology R&D.
“We are eager to bring new potential options to these patients and look forward to the results of our ongoing Phase 3 HIMALAYA trial later this year,” he added.
Also looking to make gains in the HCC market is Roche, with its own immunotherapy combination of Tecentriq (atezolizumab) and its older cancer drug Avastin (bevacizumab) recently showing a benefit as a first-line treatment for HCC.
The HCC indication is shaping up to be increasingly competitive, and important, for both Roche and AZ as the companies attempt to catch-up to immunotherapy market leader Keytruda (pembrolizumab) from Merck & Co/MSD.
AZ is also looking to beat Merck to an approval in small cell lung cancer, one of the only cancers that Keytruda has been unable to show a benefit in.
Results from the CASPIAN trial of Imfinzi in advanced small cell lung cancer revealed the drug improved overall survival by 27%. If approved, it will go on to directly compete with Roche’s Tecentriq, which is already approved in first-line SCLC.