–6th Orphan Drug Designation granted to toripalimab in the US and EU
SHANGHAI, China, July 21, 2022 (GLOBE NEWSWIRE via SEAPRWire.com) — Shanghai Junshi Biosciences Co., Ltd (“Junshi Biosciences”, HKEX: 1877; SSE: 688180), a leading innovation-driven biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies, announced today that toripalimab, a PD-1 inhibitor independently developed by Junshi Biosciences is designated as an orphan medicinal product by the European Commission (“EC”) for the treatment of nasopharyngeal carcinoma (“NPC”), following the favorable opinion of the European Medicines Agency (“EMA”). As of now, toripalimab has been granted a total of 6 Orphan Drug Designations (“ODD”) by the regulatory agencies of the European Union and the United States for the treatment of mucosal melanoma, NPC, soft tissue sarcoma, esophageal cancer, and small cell lung cancer (“SCLC”).
“We are encouraged by the ODD granted by the European Committee for toripalimab for the treatment of NPC, especially after being granted the Breakthrough Therapy Designations and ODD by the FDA,” said Dr. Patricia Keegan, Chief Medical Officer of Junshi Biosciences. “NPC is an aggressive tumor with higher incidence in Asia, thus the development of novel therapies for NPC has been slower in Europe and America. While emerging immune-oncology drugs have shown clinical efficacy in many types of tumors, none are approved for the treatment of NPC in Europe or the United States. Guided by our strategy of ‘in China, for Global,’ Junshi Biosciences spearheaded the incorporation of immunotherapy into the treatment of patients with advanced NPC in China. Now, we are eager for this innovative therapy to be available to provide benefit to patients in Europe, America, and the rest of the world in the near future!”
According to the EC, orphan medicinal products are intended for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect no more than 5 in 10,000 people in the European Union (“EU”). The EU Regulation on orphan medicinal products establishes a centralised procedure for the designation of orphan medicinal products and puts in place incentives for their research, development and marketing.
After receiving the designation, Junshi Biosciences will benefit from a number of incentives, including but not limited to: (1) protocol assistance (the EMA provides a form of scientific advice specifically for designated orphan medicines); (2) access to the centralized authorization procedure (companies are allowed to make a single applications for marketing authorization or a conditional approval to the EMA, resulting in a single opinion and a single decision from the EC, valid in all EU Member States); (3) ten years of protection from market competition once approved; (4) fee reduction for regulatory activities (including reduced fees for protocol assistance, marketing-authorization applications, inspections before authorization, application for changes to marketing authorizations made after approval, and reduced annual fees).
In the field of NPC, Junshi Biosciences has completed two pivotal clinical studies—JUPITER-02, a randomized, double blind, placebo-controlled, international multi-center Phase III clinical study, as well as POLARIS-02, a multi-center, open-label, Phase II clinical study. These studies examine first through later lines therapies in the treatment of recurrent/metastatic NPC. The JUPITER-02 results were first presented in the plenary session of the American Society of Clinical Oncology (“ASCO”) annual meeting (#LBA2) in June 2021 and subsequently published in detail as the cover article of the September 2021 issue of Nature Medicine. The POLARIS-02 results were published online in the Journal of Clinical Oncology in January 2021.
Substantiated by the results of POLARIS-02 and JUPITER-02, toripalimab has been recognized by drug regulatory agencies across the world. In 2021, two indications for the treatment of NPC were approved by the National Medical Products Administration (“NMPA”), thereby making toripalimab the world’s first immune checkpoint inhibitor approved for the treatment of NPC. The FDA has accepted for review the resubmission of the Biologics License Application (“BLA”) for toripalimab for the treatment of NPC and set the Prescription Drug User Fee Act (“PDUFA”) action date on December 23, 2022. Toripalimab will be the first and only immuno-oncology agent for NPC in U.S., if approved.
About Nasopharyngeal Carcinoma
NPC is a malignant tumor that occurs in the nasopharyngeal mucosal epithelium and is one of the most common head and neck cancers. According to the statistics of the World Health Organization, the number of newly diagnosed NPC cases in 2020 has exceeded 130,000 worldwide. Due to the location of the primary tumor, surgery is rarely an option, and patients with localized disease are treated primarily with chemotherapy and radiotherapy. In the United States and Europe, there are presently no immunotherapies approved for the treatment of NPC.
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. In China, toripalimab was the first domestic anti-PD-1 monoclonal antibody approved for marketing in 2018 (approved in China as TUOYI®). Currently, there are five approved indications for toripalimab in China.
About Junshi Biosciences
Founded in December 2012, Junshi Biosciences (HKEX: 1877; SSE: 688180) is an innovation-driven biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapeutics. The company has established a diversified R&D pipeline comprising over 50 drug candidates, with five therapeutic focus areas covering cancer, autoimmune, metabolic, neurological, and infectious diseases. Junshi Biosciences was the first Chinese pharmaceutical company that obtained marketing approval for anti-PD-1 monoclonal antibody in China. Its first-in-human anti-BTLA monoclonal antibody for the treatment of various cancers was the first in the world to be approved for clinical trials by the FDA and NMPA and has since entered Phase Ib/II trials in both China and the US. Its anti-PCSK9 monoclonal antibody was the first in China to be approved for clinical trials by the NMPA.
In the face of the pandemic, Junshi Biosciences’ response was strong and immediate, joining forces with Chinese and international scientific research institutions and enterprises to develop an arsenal of drug candidates to combat COVID-19, taking the initiative to shoulder the social responsibility of Chinese pharmaceutical companies by prioritizing and accelerating COVID-19 R&D. Among the many drug candidates is JS016 (etesevimab), China’s first neutralizing fully human monoclonal antibody against SARS-CoV-2 and the result of the combined efforts of Junshi Biosciences, the Institute of Microbiology of the Chinese Academy of Science and Lilly. JS016 administered with bamlanivimab has been granted Emergency Use Authorizations (EUA) in over 15 countries and regions worldwide. As of December 3 2021, over 700,000 patients have been treated with bamlanivimab or bamlanivimab and etesevimab, potentially preventing more than 35,000 hospitalizations and at least 14,000 deaths. Meanwhile, VV116, a new oral nucleoside analog anti-SARS-CoV-2 drug designed to hinder virus replication, is in global Phase III clinical trials. A Phase III clinical study (NCT05341609) comparing the efficacy and safety of VV116 versus nirmatrelvir/ritonavir (“PAXLOVID”) for patients with mild to moderate COVID-19 who are at high risk for progression to severe COVID-19, has reached its pre-specified primary endpoint and secondary efficacy endpoint. The study results show that compared to PAXLOVID, VV116 provided patients with a shorter median time to sustained clinical recovery, while achieving statistical superiority.The JS016 and VV116 programs are a part of the company’s continuous innovation for disease control and prevention of the global pandemic.
Junshi Biosciences has more than 2,800 employees in the United States (San Francisco and Maryland) and China (Shanghai, Suzhou, Beijing and Guangzhou). For more information, please visit: http://junshipharma.com.
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