Europe approves new immunotherapy for nasopharyngeal cancer

It is a type of head and neck cancer and affects the upper part of the throat, behind the nose. Nasopharyngeal cancer (or nasopharyngeal cancer) is the most common type of pharyngeal cancer, although it is rare. In Italy, in 2024, tumors of the upper aerodigestive tract affected a total of fewer than 6,000 people, the vast majority of whom were men (over 4,000).

Combination with immunotherapy

As you might imagine, their impact on quality of life is significant. Furthermore, nasopharyngeal cancer often cannot be operated on or treated with radiotherapy, and is discovered already in the metastatic stage due to its "deep" anatomical location and mild initial symptoms, which make early diagnosis difficult. Today's news from the European Commission concerns these cases: a new therapy—based on the combination of immunotherapy, tislelizumab, and chemotherapy—has been approved for first-line treatment.

"The approval of tislelizumab in combination with chemotherapy in Europe represents an important step forward for patients with recurrent or metastatic nasopharyngeal carcinoma, a rare and challenging disease," explains Lisa Licitra, Head of Medical Oncology 3 – Head and Neck Cancer at the Fondazione IRCCS Istituto Nazionale dei Tumori in Milan. "Thanks to the convincing results of 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 offers new hope and a clinically proven option for patients who urgently need better care."

The results of the clinical study

Currently, the 5-year survival rate for this cancer is estimated at 63% but drops to 49% when the disease is advanced. In the RATIONALE-309 clinical trial—conducted on 263 previously untreated patients—the combination of tislelizumab with gemcitabine and cisplatin significantly prolonged progression-free survival compared to gemcitabine plus cisplatin and placebo alone, reducing the risk of disease progression or death by 48%. At an additional one-year follow-up, a clinically significant and sustained improvement in overall survival was observed, reaching a median of 45.3 months in the experimental arm (i.e., half of the patients lived longer than this period) compared to 31.8 months in the control arm. Tislelizumab plus chemotherapy was generally well tolerated, and no new safety signals were identified: the most common associated serious adverse reactions were neutropenia, anemia, and thrombocytopenia.

“Following the previous EU approval of tislelizumab for extensive-stage small cell lung cancer, this new authorization for nasopharyngeal carcinoma reflects strong momentum in expanding access to our immunotherapy in solid tumors,” concluded Mark Lanasa, Chief Medical Officer, Solid Tumors at BeOne. “With a comprehensive indication in the European Union covering lung and gastrointestinal cancers and more than 100 regulatory approvals globally, we are advancing our ambition to bring innovative therapies to more patients worldwide.”