European Commission Approves First-Line Tislelizumab Plus Chemo for Metastatic Nasopharyngeal Carcinoma

Tislelizumab Plus Chemo for Metastatic

Nasopharyngeal Carcinoma | Image Credit:

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The European Commission has approved tislelizumab (Tevimbra) plus gemcitabine/cisplatin for the first-line treatment of adult patients with metastatic or recurrent nasopharyngeal carcinoma (NPC) that is not amenable to curative surgery or radiotherapy.1

This regulatory decision follows the May 2025 European Medicines Agency’s Committee for Medicinal Products for Human Use recommendation of the approval of tislelizumab plus gemcitabine/cisplatin for the respective patient population.2

The approval was based on data from the phase 3 RATIONALE-309 trial (NCT03924986), which evaluated the efficacy and safety of tislelizumab plus gemcitabine/cisplatin compared with placebo plus gemcitabine/cisplatin in treatment-naive patients with NPC (n = 263).1

“The approval of [tislelizumab] combined with chemotherapy in Europe marks an important advancement for people with recurrent or metastatic NPC—a rare and challenging disease,” Lisa Licitra, MD, chief of the Head and Neck Cancer Medical Oncology Department at Fondazione IRCCS Istituto Nazionale dei Tumori in Milan, Italy; as well as an associate professor at Università degli Studi di Milano, stated in a news release. “Thanks to the compelling results from the RATIONALE-309 study, we now have a powerful new treatment that not only delays disease progression but also helps patients live longer. This approval brings new hope and a clinically proven option to patients who urgently need better care.”

At the first prespecified interim analysis of RATIONALE-309, tislelizumab plus chemotherapy generated a significantly prolonged median progression-free survival (PFS) vs placebo plus chemotherapy in the intention-to-treat population, with a 48% reduction in the risk of disease progression or death (HR, 0.52; 95% CI, 0.38-0.73; P < .0001), meeting the trial’s primary end point. Of note, the median PFS in the tislelizumab arm (n = 131) was 9.2 months (95% CI, 7.6-10.1) vs 7.4 months (95% CI, 5.6-7.5) in the placebo arm (n = 132).3 Additionally, the independent review committee–assessed objective response rates were 69.5% vs 55.3% in the tislelizumab and placebo arms, respectively. With an additional 12 months of follow-up at the updated analysis, efficacy data were consistent with those of the interim analysis.1 Specifically, the overall survival (OS) benefit with tislelizumab plus chemotherapy vs chemotherapy alone was shown to be clinically meaningful and sustained; the median OS was 45.3 months vs 31.8 months in the tislelizumab and placebo arms, respectively.

Regarding safety, the combination of tislelizumab and gemcitabine/cisplatin was generally well tolerated without new safety signals identified. Furthermore, pooled safety data included more than 3900 patients treated with tislelizumab as monotherapy (n = 1952) or in combination with chemotherapy (n = 1950), at the approved dosing regimen. Notably, the most common grade 3/4 adverse effects (AEs) occurring in at least 10% of patients following treatment with tislelizumab plus chemotherapy included neutropenia, anemia, and thrombocytopenia.

Moreover, in RATIONALE-309, treatment-emergent AEs (TEAEs) were observed in 100.0% and 99.2% of patients from the tislelizumab and placebo arms, respectively.3 TEAEs related to tislelizumab or placebo were reported in 74.8% and 71.2% of patients from the respective arms. TEAEs leading to permanent discontinuation of any study treatment were observed in 13.0% and 9.1% of patients from the respective arms.

“Following our recent EU approval of [tislelizumab] for extensive-stage small cell lung cancer, this new authorization in NPC reflects strong momentum in broadening access to our immunotherapy across solid tumors,” Mark Lanasa, MD, PhD, chief medical officer of Solid Tumors at BeOne Medicines—the developer of tislelizumab, stated in the news release.1 “With a comprehensive European Union label spanning lung and gastrointestinal cancers, and more than 100 regulatory approvals globally, we are delivering on our ambition to bring innovative therapies to more patients around the world.”

References

1. European Commission approves Tevimbra in combination with chemotherapy as a first-line treatment for nasopharyngeal carcinoma. News release. BeOne. July 10, 2025. Accessed July 10, 2025. https://ir.beonemedicines.com/news/european-commission-approves-tevimbrar-in-combination-with-chemotherapy-as-a-first-line-treatment-for-nasopharyngeal-carcinoma/3ed59f18-6818-4ab5-8c33-3ce2b78144df
2. BeiGene receives positive CHMP opinion for Tevimbra as a first-line treatment for nasopharyngeal cancer. News release. BeiGene. May 27, 2025. Accessed July 10, 2025. https://ir.beonemedicines.com/news/beigene-receives-positive-chmp-opinion-for-tevimbrar-as-a-first-line-treatment-for-nasopharyngeal-cancer/6df997d9-f05c-4ddf-ae91-d4f2129355c1
3. Yang Y, Pan J, Wang H, et al. Tislelizumab plus chemotherapy as first-line treatment for recurrent or metastatic nasopharyngeal cancer: a multicenter phase 3 trial (RATIONALE-309). Cancer Cell. 2023;41(6):1061-1072.e4. doi:10.1016/j.ccell.2023.04.014