BeiGene (NASDAQ: BGNE) has announced that its programmed death-1 (PD-1) inhibitor, tislelizumab, has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), recommending a first market approval. The regulatory decision on whether to approve tislelizumab monotherapy for unresectable, locally advanced, or metastatic esophageal squamous cell carcinoma (ESCC) in patients previously treated with platinum-based chemotherapy is pending.
RATIONALE 302: A Global Phase III Study Supporting the MAA
The marketing authorization application (MAA) for this indication is supported by the results of the RATIONALE 302, a global Phase III study (NCT03430843) that enrolled 513 patients across 11 countries in Asia, Europe, and North America. The study met its primary endpoint, demonstrating a statistically significant and clinically meaningful survival benefit for tislelizumab compared with chemotherapy (HR 0.70 [95%CI 0.57 – 0.85]; one-sided P=.0001; median overall survival 8.6 vs 6.3 months). The safety profile was consistent with previous trials, further validating the drug’s potential for ESCC treatment.
Tislelizumab’s Growing List of Marketing Approvals in China
Tislelizumab has already garnered 11 marketing approvals in China, covering indications such as two ESCC indications, third-line classical Hodgkin’s lymphoma (cHL), metastatic urothelial carcinoma (UC), three different non-small cell lung cancer (NSCLC) indications, second-line for hepatocellular carcinoma (HCC), and unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors.
Novartis’ Licensing Deal and Development Focus
Novartis secured a USD 2 billion licensing deal with BeiGene in 2021, obtaining exclusive development rights to tislelizumab outside of China. The drug is also awaiting approval in the US for second-line ESCC treatment, with the final decision delayed due to COVID-19 induced-disruptions to on-site inspections. In November 2022, Novartis announced a strategic decision to focus the drug’s development on ESCC, canceling plans for a nasopharyngeal carcinoma (NPC) market filing and terminating NSCLC development. According to Novartis’ recent Q2 2023 financial report, the company plans to make a filing for gastric cancer and first-line ESCC use in the US this year.-Fineline Info & Tech
