China’s NMPA Approves Toripalimab in First-Line Melanoma

Melanoma | Maris– stock.adobe.com

China’s National Medical Products Administration has approved toripalimab (Tuoyi) for the frontline treatment of patients with unresectable or metastatic melanoma.1

The approval was supported by data from the phase 3 MELATORCH study (NCT03430297). Data from MELATORCH showed that patients who received the anti-PD–1 monoclonal antibody (n = 127) achieved a median progression-free survival (PFS) of 2.3 months compared with 2.1 months among patients treated with dacarbazine (n = 128; HR, 0.708; 95% CI, 0.526-0.954; P = .0209). Moreover, the median overall survival (OS) was 15.1 months vs 9.4 months, respectively (HR, 0.680; 95% CI, 0.486-0.951). The safety profile of toripalimab was consistent with previous studies, and no new safety signals were reported.

“Melanoma is a highly aggressive cancer. Due to its low sensitivity to traditional radiotherapy and chemotherapy, patients are often faced with poor survival outcomes,” Jun Guo, MD, PhD, professor and medical director, Department of Melanoma and Renal Cancer, Peking University Cancer Hospital & Institute in China, stated in a news release.

“However, thanks to melanoma’s high immunogenicity, immunotherapies such as toripalimab have significantly improved patient survival in recent years. In China, [patients with] advanced melanoma—including those in [the] second-line and later—have gained broad access to these treatments through national medical insurance. Now, toripalimab has been extended to first-line treatment of advanced melanoma. Compared to traditional chemotherapy, toripalimab has demonstrated significant advantages in PFS, overall response rate [ORR], and duration of response [DOR], as well as a clear trend toward improved OS. Notably, this approval was based on the MELATORCH study, which exclusively enrolled Chinese patients. The trial design aligned closely with clinical practice in China, and thus the findings were more relevant to Chinese [patients with] melanoma. We hope that China’s independently developed immunotherapies like toripalimab can provide a comprehensive treatment solution for advanced melanoma and offer new hope to more patients.”

MELATORCH was a multicenter, open-label, positive-controlled study that enrolled adult patients with systemic treatment-naive, histologically confirmed unresectable stage III or IV melanoma.2 Eligible patients also needed to have a measurable lesion per RECIST 1.1 criteria, and ECOG performance status of 0 or 1, adequate laboratory values, and an estimated survival of at least 16 weeks. Prior radiotherapy needed to have been completed at least 2 weeks prior to receipt of the study drug; previous adjuvant or neoadjuvant therapy was permitted but it needed to be completed at least 3 weeks prior to random assignment.

Eligible patients were randomly assigned 1:1 to receive 240 mg of intravenous (IV) toripalimab once every 2 weeks or IV dacarbazine at a dose of 1000 mg/m2 once every 3 weeks. Patients in the dacarbazine arm were permitted to cross over to the toripalimab arm in the event of disease progression.

The primary end point was PFS. Secondary end points included ORR, DOR, and treatment-related adverse effects.

Toripalimab was the first domestic anti-PD-1 monoclonal antibody approved for marketing in China.1 The agent currently holds indications in the Chinese mainland for unresectable or metastatic melanoma after failure of standard systemic therapy; recurrent or metastatic nasopharyngeal carcinoma (NPC) after failure of at least 2 lines of prior systemic therapy; locally advanced or metastatic urothelial carcinoma after disease progression on platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy; in combination with cisplatin and gemcitabine in first-line locally recurrent or metastatic NPC; in combination with paclitaxel and cisplatin in first-line unresectable locally advanced/recurrent or distant metastatic esophageal squamous cell carcinoma (ESCC); in combination with pemetrexed and platinum in first-line EGFR mutation– and ALK mutation–negative, unresectable, locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC); in combination with chemotherapy as perioperative treatment and subsequently as monotherapy for the adjuvant treatment of adult patients with resectable stage IIIA to IIIB NSCLC; in combination with axitinib (Inlyta) in first-line medium- to high-risk unresectable or metastatic renal cell carcinoma; in combination with etoposide plus platinum in first-line extensive-stage small cell lung cancer; in combination with paclitaxel for injection in first-line recurrent or metastatic triple-negative breast cancer; in combination with bevacizumab in first-line unresectable or metastatic hepatocellular carcinoma; and in first-line unresectable or metastatic melanoma.

“Within a month, toripalimab has secured approvals for two new indications—liver cancer and melanoma," Jianjun Zou, MD, PhD, general manager and chief executive officer of Junshi Biosciences added in the news release. "This milestone achievement was, without a doubt, made possible by the selfless and dedicated collaboration of researchers, participating patients, and R&D teams. Seven years ago, toripalimab had just pioneered breakthroughs in second-line melanoma treatment, becoming China’s first domestically developed anti-PD-1 monoclonal antibody and starting a new era of immunotherapy in China; today, toripalimab is again reaching new heights as it becomes the first Chinese-developed first-line immunotherapy for melanoma. Not only does this demonstrate toripalimab’s exceptional clinical value, it also reflects China’s growing strength and innovation in immuno-oncology. Moving forward, we will remain committed to advancing world-class therapies to benefit patients across the world.”

References

1. Junshi Biosciences announces the sNDA approval of toripalimab for the 1st-line treatment of melanoma. News release. Junshi Biosciences. April 25, 2025. Accessed April 25, 2025. https://www.globenewswire.com/news-release/2025/04/25/3068241/0/en/Junshi-Biosciences-Announces-the-sNDA-Approval-of-Toripalimab-for-the-1st-line-Treatment-of-Melanoma.html
2. A randomized, controlled, multi-center, phase III clinical study to investigate recombinant humanized PD-1 monoclonal antibody injection (JS001) versus dacarbazine as the 1st-line therapy for unresectable or metastatic melanoma (JS001). ClinicalTrials.gov. Updated September 19, 2024. Accessed April 25, 2025. https://clinicaltrials.gov/study/NCT03430297