First line immunotherapy for liver cancer! Imfinzi and tremelimumab, the PD-L1 inhibitors of AstraZeneca, are qualified as FDA orphan drugs!

January 21, 2020 / BIOON / -- AstraZeneca recently announced that imfinzi (trade name: yingfeifan, common name: durvalumab, duvalizumab) and tremelimumab (anti CTLA4) were granted orphan drug qualification by the US Food and Drug Administration (FDA) to treat HCC In China, imfinzi was officially approved by the National Drug Administration (nmpa) in December 2019 for the treatment of unresectable stage III non-small cell lung cancer (NSCLC) patients without disease progression after receiving platinum based chemotherapy combined with radiotherapy The results of phase III Pacific clinical trial showed that imfinzi immunotherapy after concurrent radiotherapy and chemotherapy reduced the risk of death in patients with stage III non resectable NSCLC by nearly 32%, the median progression free survival (PFS) was 16.8 months, and the total 3-year survival rate (OS) was 57% Imfinzi is the first PD-L1 immunosuppressant approved for the treatment of stage III lung cancer in mainland China This approval marks a new era of immunotherapy for stage III non-small cell lung cancer in China Orphan drug (Orphan Drug) is a kind of medicine used for prevention, treatment and diagnosis of rare diseases Rare diseases are a general term for diseases with very low incidence rate, also known as orphan diseases In the United States, rare diseases refer to the types of diseases with a population of less than 200000 Incentives for drug research and development of rare diseases include various incentives for clinical development, such as tax credits related to clinical trial costs, FDA user fee relief, FDA assistance in clinical trial design, and a 7-year market monopoly period for the approved indications after the drug is launched Liver cancer is the third leading cause of cancer death and the sixth most common cancer type in the world HCC accounts for 80% of primary liver cancer In 2018, about 700000 people in the world and 42000 in the United States were diagnosed with HCC About 80-90% of HCC patients also have chronic liver disease, which is mainly caused by hepatitis B virus (HBV) or hepatitis C virus (HCV) infection Chronic liver disease is related to inflammation Over time, inflammation leads to immunosuppression and can lead to the development of liver cancer The unique immune environment of liver cancer provides a clear theoretical basis for the study of drugs using immune system to treat liver cancer There is a critical unmet medical need for HCC patients facing limited treatment options More than half of patients are diagnosed in the late stages of the disease, usually when symptoms first appear For patients with unresectable or advanced disease, the 5-year survival rate was only 13% At present, AstraZeneca is carrying out the phase III Himalaya trial to evaluate the imfinzi monotherapy, imfinzi + tremelimumab combination therapy, and the standard treatment drug sorafenib (sorafenib, a multi kinase inhibitor), which is used for unresectable, advanced HCC patients who have not previously received systematic treatment and are not eligible for local regional treatment (limited to liver treatment) The study is being conducted in 189 clinical centers in 16 countries, including the United States, Europe, Canada, South America and Asia The primary end point is total survival (OS), and the key secondary end points include objective response rate (ORR) and progression free survival (PFS) It is worth mentioning that this study is the first to test double immunocheckpoint blockade in patients with first-line advanced HCC Jos é baselga, executive vice president of AstraZeneca's cancer research and development department, said: "many patients with liver cancer are diagnosed and treated after their condition worsens, and new effective and tolerable treatment methods are urgently needed 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 " Imfinzi is a humanized PD-L1 monoclonal antibody, which can block the combination of PD-L1 with PD-1 and CD80, thus blocking the tumor immune escape and releasing the inhibited immune response Up to now, imfinzi has been approved in 54 countries (including the United States, Japan, China and the whole European Union) for the treatment of unresectable stage III non-small cell lung cancer (NSCLC) patients without disease progression after receiving platinum based chemotherapy combined with radiotherapy In addition, imfinzi has also been approved in 11 countries, including the United States, for the treatment (previously treated) of advanced bladder cancer patients At present, the supplementary application of imfinzi combined with chemotherapy for the first-line treatment of extensive non-small cell lung cancer (es-sclc) is under the priority review of FDA in the United States The target date of PDUFA is the first quarter of 2020 Tremelimumab is a human monoclonal antibody, targeting cytotoxic T lymphocyte antigen 4 (CTLA-4), blocking the activity of CTLA-4, promoting T cell activation, initiating tumor immune response and promoting cancer cell death Tremelimumab and Bristol Myers Squibb's listed antibody drug yevoy (epimumab) target the same target CTLA-4 As a part of the new drug research and development project, imfinzi is currently exploring the therapeutic prospect of non-small cell lung cancer, small cell lung cancer, bladder cancer, head and neck cancer, liver cancer, cervical cancer, cholangiocarcinoma and other solid tumors with single drug or combination of tremelimumab and other drugs Imfinzi and tremelimumab granted orphan drug design in the US for life cancer