Sanofi Gains Chinese Approval for Two Rare Blood Disease Treatments
China's National Medical Products Administration (NMPA) has approved Sanofi's Qfitlia (fitusiran) and Cablivi (caplacizumab) for rare blood disorders. The approvals expand treatment options for hemophilia and thrombotic thrombocytopenic purpura patients.
Paris, December 11, 2025 – Sanofi announced that its treatments Qfitlia (fitusiran) and Cablivi (caplacizumab) have received approval from China's National Medical Products Administration (NMPA) for rare hematologic diseases. Qfitlia is indicated for hemophilia and Cablivi for acquired thrombotic thrombocytopenic purpura (aTTP), addressing critical unmet needs in the Chinese market.
Qfitlia is the first antithrombin-lowering therapy for the routine prophylaxis of hemophilia. Based on data from the ATLAS phase 3 studies, the treatment demonstrated significant bleed protection with a low injection frequency, suitable for patients with or without inhibitors. Hemophilia affects more than 40,000 individuals in China.
Cablivi, a Nanobody medicine, is designed to treat acquired/immune-mediated thrombotic thrombocytopenic purpura (aTTP/iTTP) in adults and adolescents aged 12 and older who weigh at least 40 kg. This rare and life-threatening disorder has a mortality rate of up to 20% with standard treatments. Cablivi targets the von Willebrand factor to inhibit microthrombi formation, thereby preventing organ damage.
The approvals of Qfitlia and Cablivi expand Sanofi's rare hematology portfolio in China. These approvals follow other recent regulatory milestones for Sanofi in the country this year, highlighting the company's commitment to bringing transformative medicines to patients with rare diseases.