China’s National Medical Products Administration (NMPA) has approved two innovative Sanofi therapies—Qfitlia (fitusiran) for hemophilia and Cablivi (caplacizumab) for acquired thrombotic thrombocytopenic purpura (aTTP/iTTP)—marking major progress in the country’s treatment landscape for rare hematologic diseases. The decisions also reinforce Sanofi’s expanding footprint in China, representing its fourth and fifth drug approvals in the country this year, following earlier authorizations for Tzield and Sarclisa across multiple indications.
Qfitlia is the first antithrombin-lowering therapy approved in China for routine prophylaxis in individuals aged 12 and older with severe hemophilia A or B, regardless of whether they have factor inhibitors. By reducing antithrombin (AT), a natural inhibitor of blood clotting, the therapy boosts thrombin generation, thereby supporting normal clot formation. The treatment uses small-interfering RNA technology, enabling infrequent dosing, low injection volume, and subcutaneous administration—an approach expected to ease the treatment burden for patients. Hemophilia affects more than 40,000 people in China, many of whom face lifelong complications and limited therapeutic options.
The approval is supported by the ATLAS phase 3 clinical program, which showed that Qfitlia delivered meaningful reductions in bleeding episodes across a broad range of patients. Key findings include a 71% reduction in annualized bleeding rates (ABR) for patients without inhibitors compared with on-demand factor therapy, and a 73% reduction for those with inhibitors compared with bypassing agents. During the open-label extension portion of the study, median ABR fell to 3.8 in patients without inhibitors and 1.9 in those with inhibitors. Nearly half of participants experienced one or no bleeds, and almost 80% required only six injections per year by the end of the extension phase.
Experts in China view Qfitlia as a milestone in modern hemophilia care. Sun Jing, Chief Physician of Hematology at Nanfang Hospital, commented that the approval ushers in a “new era of non-factor prophylactic treatment,” offering people with hemophilia A or B an alternative that reduces reliance on frequent intravenous dosing.
Sanofi’s second approval, Cablivi, is the first Nanobody-based therapy for acquired or immune-mediated thrombotic thrombocytopenic purpura in adults and adolescents weighing at least 40 kg. aTTP/iTTP is a life-threatening autoimmune disorder that causes widespread formation of micro-blood clots, leading to organ damage. Despite standard treatments such as plasma exchange and immunosuppressants, the condition carries a mortality rate of up to 20%. Around 2,700 patients in China are diagnosed with the disorder each year.
Cablivi works by targeting von Willebrand factor (vWF), a key protein involved in clot formation. By preventing vWF from binding to platelets, the therapy helps reduce the formation of harmful microthrombi. Used alongside standard care, the medicine is expected to strengthen clinical outcomes for patients at high risk of rapid deterioration.
Brian Foard, Executive Vice President and Head of Specialty Care at Sanofi, said the two approvals reflect the company’s ongoing commitment to delivering transformative therapies in China. “Qfitlia represents a potentially transformative advancement for the hemophilia community… Cablivi addresses a critical unmet need for patients facing aTTP/iTTP,” Foard said.
While Qfitlia offers significant clinical benefits, its label includes warnings for thrombotic events, hepatotoxicity, and gallbladder disease. The most common adverse reactions include viral infections, nasopharyngitis, and bacterial infections.
Together, the approvals of Qfitlia and Cablivi mark an important expansion of Sanofi’s rare disease portfolio in China, offering long-awaited treatment options for patients with both chronic bleeding disorders and acute clotting emergencies.