Sanofi Secures Chinese Approval for Two Rare Blood Disorder Therapies Qfitlia and Cablivi
核心洞察
China's National Medical Products Administration approved Sanofi's Qfitlia (搜索) (fitusiran) for hemophilia prophylaxis, marking the first antithrombin-lowering therapy that requires as few as six injections annually.
The agency also approved Cablivi (caplacizumab), the first Nanobody medicine targeting acquired thrombotic thrombocytopenic purpura, a rare life-threatening blood clotting disorder affecting approximately 2,700 Chinese patients annually.
Clinical data from the ATLAS program demonstrated Qfitlia (搜索) reduced bleeding rates by 71% in patients without inhibitors and 73% in those with inhibitors compared to on-demand treatment.
China's National Medical Products Administration (NMPA) has approved two groundbreaking Sanofi therapies for rare blood disorders, bringing innovative treatment options to thousands of patients with previously limited therapeutic choices. The December 11, 2025 approvals of Qfitlia (搜索) (fitusiran) for hemophilia and Cablivi (caplacizumab) for acquired thrombotic thrombocytopenic purpura represent significant advances in rare hematology care.
Revolutionary Hemophilia Treatment Reduces Injection Burden
Qfitlia (搜索) emerges as the first antithrombin-lowering therapy for routine prophylaxis in hemophilia patients, offering a paradigm shift from traditional factor replacement therapy. The treatment is indicated for pediatric patients 12 years and older and adults with severe hemophilia A or B, with or without factor inhibitors.
The therapy's most striking feature is its dosing convenience, requiring as few as six subcutaneous injections annually. This represents a dramatic reduction from traditional factor therapies that often require multiple weekly infusions. By lowering antithrombin, a protein that inhibits blood clotting, Qfitlia (搜索) helps increase thrombin generation to restore hemostasis in people with hemophilia.
"The approval of Qfitlia (搜索) marks a true transition into a new era of non-factor prophylactic treatment for hemophilia in China," said Sun Jing, Chief Physician of Hematology at Nanfang Hospital, Southern Medical University, Guangzhou. "Requiring potentially just six subcutaneous injections annually, it significantly reduces disease burden, eliminating the need for frequent intravenous injections associated with traditional factor therapy."
Compelling Clinical Evidence from ATLAS Program
The ATLAS phase 3 clinical development program provided robust evidence supporting Qfitlia (搜索)'s efficacy. Key findings demonstrated significant bleeding reduction across patient populations:
- 71% reduction in annualized bleeding rates (ABR) for patients without inhibitors compared to on-demand clotting factor concentrate (9.0 vs. 31.4, p<0.0001)
- 73% reduction in ABR for patients with inhibitors compared to bypassing agent on-demand (5.1 vs. 19.1, p<0.0006)
- Median observed ABR during open-label extension was 3.8 in patients without inhibitors and 1.9 in those with inhibitors
- Nearly half of patients experienced one or fewer bleeds during the extension study (47% had 0-1 bleeds, 31% had zero bleeds)
- 80% of participants achieved a six-injection-per-year regimen by study conclusion, with 94% reaching target antithrombin levels with minimal dose adjustments
First Nanobody Therapy for Life-Threatening Blood Clotting Disorder
Cablivi addresses an equally critical unmet need as the first Nanobody medicine designed to treat acquired/immune-mediated thrombotic thrombocytopenic purpura (aTTP/iTTP) in adults and adolescents aged 12 or older weighing at least 40 kg. This ultra-rare autoimmune blood clotting disorder affects approximately 2,700 patients annually in China and carries a mortality rate of up to 20% despite standard treatments.
The therapy targets von Willebrand factor (vWF), a protein involved in hemostasis, and inhibits the interaction between vWF and platelets. Used alongside plasma exchange and immunosuppressive therapy, Cablivi helps prevent the formation of microthrombi that contribute to organ damage during disease episodes.
Addressing Substantial Patient Populations
These approvals address significant patient populations in China, with hemophilia affecting more than 40,000 people nationwide. The conditions represent substantial unmet medical needs, as hemophilia patients face lifelong bleeding risks that can result in joint damage and chronic pain, while aTTP patients confront a life-threatening emergency requiring immediate intervention.
"Qfitlia (搜索) represents a potentially transformative advancement for the hemophilia community in China, shifting care from treating bleeds as they occur to helping prevent them altogether," said Brian Foard, Executive Vice President, Head of Specialty Care at Sanofi. "Cablivi addresses a critical unmet need for patients facing aTTP/iTTP. Together, these approvals highlight Sanofi's commitment to delivering meaningful innovation and improving outcomes for people living with rare diseases in China and around the world."
Safety Considerations and Technology Platform
Qfitlia (搜索) utilizes small-interfering RNA technology with Alnylam Pharmaceutical (搜索)'s ESC-GalNAc conjugate technology, enabling its low treatment frequency and subcutaneous administration. However, serious thrombotic events, acute and recurrent gallbladder disease, and hepatotoxicity have occurred in treated patients. The most common adverse reactions include viral infection, nasopharyngitis, and bacterial infection.
Cablivi represents the first Nanobody VHH technology approved for this indication and is currently available in nearly 30 countries. The therapy earned priority review status in China, reflecting the urgent medical need it addresses.
Global Regulatory Progress
The Chinese approvals follow Qfitlia (搜索)'s March 28, 2025 FDA approval in the United States for similar indications. Additional regulatory submissions are under review worldwide, suggesting broader global access may follow. These approvals mark Sanofi's fourth and fifth approvals in China for 2025, following Tzield for type 1 diabetes and Sarclisa for multiple myeloma indications.