AstraZeneca has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for Imfinzi (durvalumab) to treat hepatocellular carcinoma (HCC), a common form of liver cancer.
Imfinzi is a monoclonal antibody designed to bind to and inhibit the interaction of PD-L1 with PD-1 and CD80. The drug is said to block the tumour’s immune-evading mechanism and release the inhibition of the immune response.
Currently, the drug has approval in 54 countries in the curative-intent setting of unresectable, stage III non-small cell lung cancer (NSCLC) following chemoradiation. It is also approved in 11 countries for previously treated patients suffering from advanced bladder cancer.
The drug previously secured the FDA priority review designation for use in combination with chemotherapy in treatment naïve extensive-stage small cell lung cancer (SCLC).
Furthermore, the FDA has now granted ODD to tremelimumab, which blocks cytotoxic T-lymphocyte-associated protein 4 (CTLA-4) activity.
The company is studying Imfinzi and tremelimumab combination in NSCLC, bladder cancer, SCLC, head and neck cancer and liver cancer.
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.”
HIMALAYA is being conducted at 189 sites in 16 countries to evaluate Imfinzi and Imfinzi / tremelimumab combination in unresectable, advanced HCC. The trial involves patients who did not receive prior systemic therapy and are ineligible for locoregional treatment.
The primary endpoint is overall survival, while significant secondary outcomes include objective response rate and progression-free survival.
AstraZeneca, along with its partner MSD (Merck), has also obtained priority review designation for Lynparza (olaparib) in metastatic castration-resistant prostate cancer (mCRPC) patients with deleterious or suspected deleterious germline or somatic homologous recombination repair (HRR) gene mutations.