FDA Approves BeiGene’s Tevimbra for Unresectable or Metastatic Esophageal Squamous Cell Carcinoma

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The FDA has approved BeiGene’s Tevimbra (tislelizumab-jsgr) in combination with platinum-containing chemotherapy as a first-line treatment for adults with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) whose tumors express PD-L1 (≥1). The approval was based on data from the Phase III RATIONALE-306 trial, which demonstrated a significant overall survival (OS) benefit in patients administered the monoclonal antibody.¹

“The approval of Tevimbra in combination with chemotherapy for adult patients with ESCC expands first-line treatment options for patients with this disease,” said Nataliya Uboha, associate professor, University of Wisconsin, Carbone Cancer Center, in a press release. “There is a critical need for effective treatments of ESCC, and Tevimbra has been shown to improve outcomes in this patient population.”

The randomized, placebo-controlled, double-blind, global RATIONALE-306 study evaluated the safety and efficacy of the Tevimbra-based combination treatment in 649 adults with a primary endpoint of OS. Patients were randomly assigned in a 1:1 ratio to receive Tevimbra or placebo intravenously every three weeks with an investigator-chosen chemotherapy, including a platinum agent plus a fluoropyrimidine or capecitabine. Treatment was continued until disease progression or unacceptable toxicity.^1,2

Results demonstrated a median OS of 16.8 months for patients in the Tevimbra group and 9.6 months for patients in the placebo group, reflecting a 34% reduction in the risk of death. Serious adverse events (AEs) included pneumonia, dysphagia, diarrhea, fatigue, and esophageal stenosis. The most common AEs included anemia; fatigue; decreased appetite; nausea; constipation; decreased weight; diarrhea; peripheral sensory neuropathy; vomiting; and stomatitis. The safety profile of Tevimbra was previously evaluated in the global RATIONALE-306 trial and no new signals were reported.¹

According to the American Cancer Society, esophageal cancer is significantly more common in men than in women, with statistics indicating that men have a one in 127 chance of developing the disease compared to one in 434 for women. It is estimated that by the end of 2025, there will be approximately 22,070 newly diagnosed esophageal cancer cases with 16,250 deaths in the United States. It accounts for 1% of all cancers in the United States, but is more common in areas such as Iran, northern China, India, and southern Africa. ESCC is most common in African Americans, but esophageal cancer overall is more common in White people.³

According to the National Cancer Institute, around four in every 100,000 people will be diagnosed with esophageal cancer in the United States annually. Adenocarcinoma is the most common subtype in the United States, accounting for 80% of all cases. Past research has indicated that 9% of all cases can be traced back to family history of Barrett’s esophagus, which has been linked to esophageal adenocarcinoma.⁴

According to BeiGene, ESCC is the sixth most common cause of cancer-related deaths globally and accounts for around 90% of all esophageal cancers. By 2040, it is expected that there will be around 957,000 new cases.¹

“Today’s approval, our third from the FDA in less than a year, reflects our dedication to advancing innovative therapies and addressing critical needs in cancer care,” said Mark Lanasa, MD, PhD, chief medical officer, solid tumors, BeiGene, in the press release. “FDA approval of Tevimbra for the first-line treatment of advanced esophageal squamous cell carcinoma marks a significant step forward in tackling the unmet needs in this challenging disease area. We are grateful to the patients, clinicians, and researchers whose commitment and courage have made this progress possible.”

References

1. TEVIMBRA Approved in U.S. for First-line Treatment of Advanced Esophageal Squamous Cell Carcinoma in Combination with Chemotherapy. BeiGene. March 4, 2025. Accessed March 4, 2025. https://ir.beigene.com/news/tevimbra-approved-in-u-s-for-first-line-treatment-of-advanced-esophageal-squamous-cell-carcinoma-in-combination/8379a7c3-35ce-45af-82d3-164c64ecf37c/

2. Tislelizumab plus chemotherapy versus placebo plus chemotherapy as first-line treatment for advanced or metastatic esophageal squamous cell carcinoma (RATIONALE-306): a global, randomized, placebo-controlled, phase 3 study. The Lancet. Accessed March 4, 2025. https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(23)00108-0/abstract

3. Key Statistics for Esophageal Cancer. American Cancer Society. Accessed March 4, 2025. https://www.cancer.org/cancer/types/esophagus-cancer/about/key-statistics.html

4. Esophageal Cancer. NIH. Accessed March 4, 2025. https://www.cancer.gov/pediatric-adult-rare-tumor/rare-tumors/rare-digestive-system-tumors/esophageal#:~:text=Esophageal%20cancer%20is%20a%20rare,coughed%20up%20from%20the%20lungs