The FDA approved toripalimab (Loqtorzi) on October 27, 2023, as the first immunotherapy for advanced nasopharyngeal carcinoma (NPC), an aggressive head and neck cancer tied to Epstein-Barr virus (EBV), genetic predispositions, and various environmental risks. Early detection and chemoradiation offered hope, but advanced or recurrent cases historically lacked effective therapies—until toripalimab’s bridged this gap through a strategic evidence journey.

Building Clinical Evidence

The toripalimab evidence journey began with clinical trials in Asia. The JUPITER-02 trial showed toripalimab plus chemotherapy extended median progression-free survival (PFS) to 21.4 months versus 8.2 months with chemotherapy alone (HR 0.52, P < 0.0001), with two-year overall survival (OS) rates of 78% versus 65%.

The POLARIS-02 trial confirmed efficacy in pretreated patients, with a 20.5% objective response rate and median OS of 17.4 months. These results established toripalimab as a first-line and salvage therapy, marking it as the first FDA-approved PD-1 inhibitor for NPC and signaling a potential shift to immunotherapy as standard care.

Emergent Economic Evidence

As toripalimab’s clinical benefits took shape, studies began exploring its cost advantages over other immunotherapies like pembrolizumab and nivolumab. Research showed it delivers similar survival improvements—outpacing chemotherapy alone—but at a lower price. In the U.S., it proved affordable for healthcare payers, costing far less than typical immunotherapy thresholds . In China, its lower price as a locally made drug made it a practical option compared to other immunotherapies. Notably, pembrolizumab and nivolumab required steep discounts to compete. U.S. analyses even projected savings of $174–184 million over pembrolizumab. This evidence showed that toripalimab is a budget-friendly choice that could ease financial pressures on healthcare systems.

Conclusion: The Triumph of Dual Evidence