FDA Accepts AstraZeneca BLA for Hepatocellular Carcinoma Antibody

FDA recently accepted AstraZeneca’s biologics license application (BLA) for its anti-CTLA4 antibody, tremelimumab, supporting the indication of a single primary dose of the drug added to Imfinzi to treat patients with unresectable hepatocellular carcinoma (HCC).  

FDA based the BLA for tremelimumab and sBLA for Imfinzi on the HIMALAYA Phase 3 trial, which found that patients treated with the dosing schedule experienced a 22% reduction in the risk of death versus sorafenib. 

Additionally, nearly one in three patients (31%) were still alive at three years versus one in five (20%) for sorafenib.  

“The HIMALAYA Phase III trial showed an unprecedented three-year overall survival in this setting with a single priming dose of tremelimumab added to Imfinzi, highlighting the potential for this regimen to improve longer-term survival outcomes,” Susan Galbraith, executive vice president of oncology research and development at AstraZeneca, said in the announcement.  

“Patients with advanced liver cancer are in great need of new treatment options, and we are working closely with the FDA to bring this novel approach to patients in the US as soon as possible,” Galbraith continued.  

Liver cancer, of which HCC is the most common type, is the third-leading cause of cancer death and the sixth most commonly diagnosed cancer globally. Nearly 26,000 people in the US present with advanced, unresectable HCC each year.  

In January 2020, FDA granted Imfinizi and tremelimumab orphan drug designation to treat HCC.  

Then in January 2022, AstraZeneca announced that Imfinzi combined with standard-of-care chemotherapy significantly improved overall survival and progression-free survival in patients with advanced biliary tract cancer (BTC). 

In the study, patients who received the combination treatment experienced a 20% reduction in the risk of death versus chemotherapy alone and a 25% reduction in risk of disease progression or death with Imfinzi plus chemotherapy.  

The median overall survival for these patients was 12.8 months versus 11.5 months for chemotherapy alone. And about 25% of patients were still alive at two years, compared to 10% of patients who just received chemotherapy. 

AstraZeneca will continue to assess Imfinzi across multiple liver cancer settings, including locoregional HCC and adjuvant HCC.