BeiGene’s Tislelizumab Receives 12th Regulatory Approval in China: A Step Closer to Cancer Treatment Breakthrough

The submission to regulatory authorities is a crucial step towards obtaining marketing authorization for the use of a new treatment in the fight against unresectable or metastatic hepatocellular carcinoma. This is the first time such a treatment has been proposed for first-line treatment, providing a much-needed advancement in the treatment of this devastating cancer.

BeiGene, a global biotechnology leader, has just received exciting news: The China National Medical Products Administration (NMPA) has accepted a supplemental biologics license application (sBLA) for tislelizumab, a innovative treatment for unresectable or metastatic hepatocellular carcinoma (HCC). This is a major step forward in providing a potentially life-saving therapy for patients suffering from this devastating form of cancer.

Hepatocellular Carcinoma (HCC) is a devastating form of primary liver cancer that affects people around the world. In China, the situation is particularly dire, with half of all new HCC cases and deaths occurring in the country. Sadly, the 5-year survival rate for those affected by HCC in China stands at only 14%, a stark reminder of the gravity of this disease.

BeiGene is enthusiastic to collaborate with the NMPA on the submission of its rigorously studied global clinical development program for tislelizumab in HCC. Despite the rising incidence of HCC in China, the treatment landscape has not seen much advancement, with modest survival benefits and sub-optimal tolerability of multi-kinase inhibitors. BeiGene is confident that the efficacy and favorable tolerability profile showcased in tislelizumab will bring a much-needed new treatment option to HCC patients in China.

The RATIONALE 301 clinical trial (NCT03412773) was a groundbreaking endeavor, enrolling 674 patients from research centers across Asia, Europe, and the United States. The results of the trial were presented at the 2022 European Society for Medical Oncology (ESMO) Congress in Paris as a late-breaking oral presentation, and the success of the trial has led to the sBLA being supported.

Tislelizumab has been approved by the China NMPA for a range of nine indications, including conditional approval for the treatment of hepatocellular carcinoma (HCC). In addition, the China Drug Evaluation (CDE) is currently reviewing additional sBLAs for tislelizumab, such as combination with chemotherapy as a first-line treatment for advanced or metastatic gastric or gastroesophageal junction adenocarcinoma, and combination with chemotherapy as first-line treatment for unresectable locally advanced, recurrent or metastatic esophageal squamous cell carcinoma. Unfortunately, this groundbreaking drug is not yet available for use outside of China.

About Tislelizumab

Tislelizumab is a special humanized IgG4 anti-PD-1 monoclonal antibody that has been specifically designed to limit its binding to macrophages’ Fc-gamma (Fcγ) receptors. This is to ensure that the body’s immune cells are able to detect and fight tumors more effectively. Pre-clinical studies have revealed that too much binding to Fcγ receptors on macrophages can compromise the anti-tumor activity of PD-1 antibodies by activating the so-called antibody-dependent macrophage-mediated killing of T effector cells.

Tislelizumab, the first investigational medicine from BeiGene’s immuno-oncology biologics program, is being tested to treat solid tumor and hematologic malignancies both on its own and in combination with other treatments. To date, the global tislelizumab clinical development program has enrolled over 11,500 participants from more than 30 countries and regions in 21 registration-enabling trials. With its promising potential, tislelizumab is a promising treatment for many different kinds of cancers.


The Phase 3, global, randomized, open-label NCT03412773 study is assessing the efficacy of tislelizumab compared to sorafenib as a first-line treatment for adult patients with unresectable HCC. The primary endpoint of this study is to determine the non-inferiority of Overall Survival between the two treatment groups. The key secondary endpoint is the Overall Response Rate as assessed by Blinded Independent Review Committee (BIRC) per RECIST v1.1. In addition, the study will also evaluate other efficacy assessments such as Progression Free Survival, Duration of Response, and Time to Progression per BIRC, as well as measures of health-related quality of life, and safety and tolerability.

About BeiGene

At BeiGene, we are revolutionizing the way oncology treatments are accessed and experienced by millions of patients around the world. Our passionate team of over 9,000 individuals spanning five continents are dedicated to developing and commercializing innovative, affordable medicines that will improve outcomes and expand access. Our diverse pipeline of novel therapeutics is being expedited through our internal capabilities and collaborations, with administrative offices located in Cambridge, U.S., Basel, Switzerland, and Beijing, China. Join us in our mission to make life better for those affected by cancer.

Forward-Looking Statements

BeiGene is excited to announce the potential of tislelizumab to treat patients with HCC, and is committed to advancing its anticipated clinical development, regulatory milestones and eventual commercialization of tislelizumab. However, there are certain risks and uncertainties that could affect BeiGene’s success, such as the ability to demonstrate the efficacy and safety of its drug candidates and the impact of the COVID-19 pandemic on BeiGene’s clinical development, regulatory, commercial, manufacturing, and other operations. Nonetheless, BeiGene is confident in its abilities to obtain and maintain protection of intellectual property for its medicines and technology, obtain additional funding for operations, and achieve and maintain profitability, as it pursues its plans, commitments, aspirations and goals.

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