On January 25, 2021 Junshi Biosciences (HKEX: 1877; SSE: 688180), an innovation-driven biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies, reported that U.S. Food and Drug Administration (FDA) has granted toripalimab Fast Track designation for the first-line treatment of mucosal melanoma (Press release, Shanghai Junshi Bioscience, JAN 25, 2021, View Source [SID1234574255]). Meanwhile, the FDA has also approved the Investigational New Drug (IND) application for a global Phase III trial of Toripalimab in combination with Axitinib versus Pembrolizumab for the first-line treatment of patients with advanced mucosal melanoma (Combination Clinical Trial).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
About Toripalimab
Toripalimab was the first domestic anti-PD-1 monoclonal antibody approved for marketing in China. More than thirty company-sponsored clinical studies covering more than fifteen indications have been conducted globally, including in China and the United States. On December 17, 2018, Toripalimab obtained a conditional approval from the National Medical Products Administration (the "NMPA") for the second-line treatment of unresectable or metastatic melanoma. Toripalimab was included in the 2019 and 2020 Guidelines of Chinese Society of Clinical Oncology (CSCO) for the Diagnosis and Treatment of Melanoma. Two supplemental New Drug Applications (NDAs) of Toripalimab for the third-line treatment of recurrent/metastatic nasopharyngeal carcinoma and the second-line treatment of metastatic urothelial carcinoma were accepted by the NMPA in April and May 2020, respectively. Both supplemental NDAs received priority review designations from the NMPA in July 2020. In addition, Toripalimab for the treatment of recurrent/metastatic nasopharyngeal carcinoma was granted the Breakthrough Therapy Designation (BTD) by the FDA in September 2020. In December 2020, Toripalimab was included in the updated National Reimbursement Drug List (NRDL) (2020 Edition).
About Combination Clinical Trial
The Combination Clinical Trial is an international, multi-center, randomized Phase III study designed to evaluate the efficacy and safety of Toripalimab in combination with Axitinib versus Pembrolizumab as a first-line treatment in patients with unresectable, locally advanced or metastatic mucosal melanoma. The trial intends to enroll 220 patients who will be randomized at 1:1 ratio into two study arms. The primary endpoint of the Combination Clinical Trial is progression-free survival (PFS). The secondary endpoints include objective response rate (ORR), duration of response (DOR), overall survival (OS), and safety.
Mucosal melanoma is a subtype of melanoma with mucosal origin. Compared with cutaneous melanoma, mucosal melanoma is less responsive to traditional chemotherapy and immunotherapy, and thus remains an unmet medical need. Toripalimab and Axitinib combination was the world’s first clinical application of PD-1 checkpoint blockade with a VEGFR inhibitor for the treatment of mucosal melanoma in a Phase Ib study (NCT03086174). The results of the study were published in the Journal of Clinical Oncology in August 2019. The study showed that the combination of Toripalimab with Axitinib in the first-line setting achieved an ORR of 48.3% and a disease control rate (DCR) of 86.2% for advanced mucosal melanoma with a manageable safety profile. The median progression-free survival (mPFS) was 7.5 months. In March 2020, Toripalimab in combination with Axitinib for the treatment of mucosal melanoma was granted the Orphan-Drug Designation (ODD) by the FDA.
About Fast Track Designation (FTD)
The FTD aims to facilitate the development and accelerate the review process of treatments for serious or life-threatening diseases or conditions that remain unmet clinical needs. The FTD is designed to make important new drugs accessible to patients faster. According to FDA regulations, drugs that receive FTDs will be granted an accelerated review process through various forms, including but not limited to (1) more frequent interactions and meetings with the FDA to discuss drug development and clinical trial design; (2) eligibility for priority review and accelerated approval after meeting relevant criteria; (3) rolling review, which means instead of waiting until all sections of the NDA are completed, a company can submit completed sections of its biologic license application (BLA) or new drug application (NDA) for review on a rolling basis.
The FTD will significantly accelerate the research, development, and marketing of Toripalimab in the United States. This marks another important milestone, after Toripalimab was granted the ODD and the BTD by the FDA.