Shanghai Junshi Biosciences Co., Ltd. announced that the National Medical Products Administration (NMPA) has approved the supplemental new drug application (sNDA) for toripalimab (product code:JS001) in combination with etoposide plus platinum for the first-line treatment of extensive-stage small cell lung cancer (ES-SCLC). Lung cancer is currently the most prevalent malignant tumor with the highest mortality rate in China. However, most patients have already been diagnosed with ES-SCLC when seeking medical treatment, with a median survival time of less than one year and a two-year survival rate of less than 10%, and thus ES-SCLC remains a major unmet clinical problem.

The approval of the supplemental new drug application is mainly based on data from the EXTENTORCH study (NCT04012606), a randomized, double-blind, placebo-controlled, multi-center Phase 3 clinical study, aiming to compare the efficacy and safety of toripalimab or placebo in combination with etoposides plus platinum for the first- line treatment of ES-SCLC. Led by principal investigator Professor Ying CHENG from Jilin Cancer Hospital, the study was conducted across 51 clinical centers nationwide. At the European Society for Medical Oncology (ESMO) Congress held in October 2023, the EXTENTORCH data was released in the form of an oral presentation for the first time.

The study results showed that, compared to chemotherapy alone, toripalimab in combination with chemotherapy could significantly prolong the PFS and OS of patients and demonstrated a favorable safety profile. This suggests that toripalimab may be an optimal immunotherapy for ES-SCLC. In particular, the median PFS of the toripalimab group reached 5.8 months, and the risk of disease progression or death was reduced by 33.3% (P=0.0002).

The one-year PFS was nearly four times higher in the toripalimab group compared with the chemotherapy group (18.1% vs. 4.9%). The median OS of the toripalimab group reached 14.6 months, the risk of death was reduced by 20.2% (P=0.0327), and the one-year OS reached 63.1%.

Toripalimab is an anti-PD-1 monoclonal antibody developed for its ability to block PD-1 interactions with its ligands, PD-L1 and PD-L2, and for enhanced receptor internalization (endocytosis function). Blocking PD-1 interactions with PD-L1 and PD-L2 promotes the immune system?s ability to attack and kill tumor cells. More than forty company-sponsored toripalimab clinical studies covering more than fifteen indications have been conducted globally by Junshi Biosciences, including in China, the United States, Southeast Asia, and Europe.

Ongoing or completed pivotal clinical trials evaluating the safety and efficacy of toripalimab cover a broad range of tumor types, including cancers of the lung, nasopharynx, esophagus, stomach, bladder, breast, liver, kidney, and skin. In Chinese mainland, toripalimab was the first domestic anti-PD-1 monoclonal antibody approved for marketing (approved in China as TUOYI®). Currently, there are nine approved indications for toripalimab in Chinese mainland: unresectable or metastatic melanoma after failure of standard systemic therapy; recurrent or metastatic nasopharyngeal carcinoma (NPC) after failure of at least two lines of prior systemic therapy; locally advanced or metastatic urothelial carcinoma that failed platinum-containing chemotherapy or progressed within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy; in combination with cisplatin and gemcitabine as the first-line treatment for patients with locally recurrent or metastatic NPC; in combination with paclitaxel and cisplatin in first-line treatment of patients with unresectable locally advanced/recurrent or distant metastatic esophageal squamous cell carcinoma (ESCC); in combination with pemetrexed and platinum as the first-line treatment in EGFR mutation-negative 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 with monotherapy as adjuvant therapy for the treatment of adult patients with resectable stage IIIA-IIIB NSCLC; in combination with axitinib for the first-line treatment of patients with medium to high risk unresectable or metastatic renal cell carcinoma (RCC); in combination with etoposide plus platinum for the first-line treatment of extensive-stage small cell lung cancer (ES-SCLC).

The first six indications have been included in the National Reimbursement Drug List (NRDL) (2023 Edition). Toripalimab is the only anti-PD-1 monoclonal antibody included in the NRDL for the treatment of melanoma. In April 2024, the Drug Office at the Department of Health in the Government of the Hong Kong Special Administration Region (DO) accepted the NDA for toripalimab in combination with cisplatin and gemcitabine for the first-line treatment of adults with metastatic or recurrent locally advanced NPC, and for toripalimab, as a single agent, for the treatment of adults with recurrent, unresectable, or metastatic NPC with disease progression on or after platinum-containing chemotherapy.

In Singapore, the NDA application was accepted by the Singapore Health Sciences Authority (HSA) in January 2024. The HSA has also granted priority review designation for the NDA.