Novo Nordisk Beats CSPC to File Recombinant Factor VIII for Market Approval in China

Author: Cai Cai

Recently, Novo Nordisk’s recombinant coagulation factor VIII (turoctocog alfa, brand name: Novoeight) was submitted for marketing approval in China for the treatment of hemophilia A. Previously, Chia Tai Tianqing had completed Phase III clinical trials ahead of Novo Nordisk; however, Novo Nordisk filed its marketing application with the National Medical Products Administration (NMPA) before Chia Tai Tianqing.

(Data source: Yaozhi Data)

Turoctocog alfa is a B-domain-deleted recombinant human coagulation factor VIII used for the prevention and treatment of bleeding episodes in patients with hemophilia A. As Novo Nordisk’s third-generation recombinant coagulation factor VIII product, it leverages state-of-the-art gene recombination and protein purification technologies and was approved by the FDA in October 2013.

Turoctocog alfa was evaluated in the Phase III Guardian program, the largest pre-licensure clinical trial program conducted to date in patients with hemophilia A, involving more than 200 patients with hemophilia A. These Phase III clinical trials, conducted by Novo Nordisk, included previously treated adult and pediatric patients with severe hemophilia A. The study results demonstrated the efficacy of turoctocog alfa in both prophylaxis and treatment of bleeding episodes, with no inhibitor development observed in patients.

Currently, the global annual sales of Novo Nordisk’s Novoeight and Novoseven are approximately $2 billion.

Long-Acting Recombinant Coagulation Factors Become the Future R&D Trend

Hemophilia is a group of rare hereditary hemorrhagic disorders characterized by impaired coagulation. Its common features include defective generation of active thromboplastin, prolonged clotting time, and a lifelong tendency to bleed after minor trauma. In severe cases, "spontaneous" bleeding may occur even in the absence of significant external injury. Based on the specific deficient clotting factor, hemophilia is typically classified into types A, B, and C. Hemophilia A, caused by a deficiency of clotting factor VIII, accounts for 80%–85% of all cases.

(Source: CNKI)

Hemophilia is inherited in an X-linked recessive pattern. The gene is carried for life, and there is currently no cure. Current treatments are all replacement therapies, with clotting factors being the primary medications for treating hemophilia. Among these, Factor VIII is a glycoprotein with a molecular weight exceeding one million daltons. It dissociates into subunits with a molecular weight of approximately 200,000 daltons under high-salt conditions. As a cofactor for Factor IXa, it participates in the activation of Factor X during the coagulation cascade and is primarily used to treat Hemophilia A.

To date, hemophilia replacement therapies have evolved through three generations, with long-acting recombinant coagulation factors representing the future trend in research and development.

(Compiled from publicly available information)

Foreign Pharmaceutical Companies Hold Nearly 70% of the Coagulation Factor VIII Market Share

Coagulation Factor VIII products currently marketed in China include first-, second-, and third-generation formulations. Domestic pharmaceutical companies primarily offer first-generation products, whereas foreign pharmaceutical companies provide second- and third-generation products. Notably, Roche’s emicizumab (Emicizumab Injection), which was approved for marketing in China at the end of last year, has attracted significant attention. Emicizumab is a recombinant humanized bispecific monoclonal antibody that received FDA approval in the United States at the end of 2017. Compared with other hemophilia therapies, it has a prolonged duration of action and requires only once-weekly subcutaneous administration. It is currently the only long-acting recombinant coagulation factor VIII product available on the Chinese market.

(Compiled from public sources)

Currently, in the domestic market for coagulation factor VIII, foreign pharmaceutical companies account for nearly 70% of the market share.

Novo Nordisk Beats Chia Tai Tianqing to File for Marketing Approval, but Chia Tai Tianqing’s Product is Long-Acting

Currently, pharmaceutical companies both domestically and internationally are actively positioning recombinant coagulation factor VIII in China. The following companies are in Phase III clinical trials, with Novo Nordisk taking the lead over Chia Tai Tianqing in filing for market approval.

Notably, both Furen Pharmaceutical and Chia Tai Tianqing’s products are third-generation long-acting recombinant human coagulation factor VIII-Fc fusion proteins, which offer significant advantages over Novo Nordisk’s second-generation product.

(Source: CDE, Company Announcements)

Theoretical Market Size of Coagulation Factor VIII in China Exceeds RMB 10 Billion

According to PDB data, sales of coagulation factor VIII in sample hospitals increased from RMB 130 million in 2012 to RMB 466 million in 2017, representing a compound annual growth rate (CAGR) of 29.15%. Extrapolating based on a multiplier of 5–6 times the 2017 sample hospital sales figures, the current market size for coagulation factor VIII is approximately RMB 2.33 billion to RMB 2.796 billion (average: RMB 2.563 billion). Some securities analysts estimate that, given a potential population of 141,300 patients with hemophilia A in China, the theoretical market potential for coagulation factor VIII exceeds RMB 16.8 billion.

Title: Filing | Novo Nordisk’s Recombinant Coagulation Factor VIII Submits for Market Approval in China, Outpacing Chia Tai Tianqing! Hemophilia’s Blue-Ocean Market Exceeds RMB 10 Billion