China’s National Medical Products Administration (NMPA) has greenlit two innovative Sanofi therapies for rare hematologic diseases.
With these approvals, Sanofi strengthens its rare hematology portfolio in China, tackling both chronic bleeding disorders and acute clotting emergencies while advancing its long-term commitment to bringing transformative medicines to Chinese patients.
The approvals for Qfitlia (fitusiran) for hemophilia and Cablivi (caplacizumab) for acquired thrombotic thrombocytopenic purpura (aTTP) mark the global biopharma giant's fourth and fifth wins in China this year, following Tzield for stage 2 type 1 diabetes and Sarclisa for multiple myeloma.
“Qfitlia represents a potentially transformative advancement for the hemophilia community in China, shifting care from treating bleeds as they occur to helping prevent them altogether. By offering effective bleed protection and simplified administration, Qfitlia has the potential to make prophylaxis more accessible for people with hemophilia worldwide,” said Brian Foard, Executive Vice President, Head of Specialty Care, Sanofi.
Qfitlia is the first antithrombin (AT)-lowering therapy approved for routine prophylaxis in hemophilia, aimed at pediatric patients 12 and older, as well as adults with severe hemophilia A or B, with or without inhibitors. Clinical data from the ATLAS phase 3 studies showed dramatic reductions in annualized bleeding rates (ABR), with prophylaxis cutting bleeds by 71% in patients without inhibitors and 73% in those with inhibitors.
“The approval of Qfitlia marks a true transition into a new era of non-factor prophylactic treatment for hemophilia in China. Requiring potentially just six subcutaneous injections annually, it significantly reduces disease burden, eliminating the need for frequent intravenous injections associated with traditional factor therapy,” said Sun Jing, Chief Physician of Hematology, Nanfang Hospital at Southern Medical University, Guangzhou.
“By lowering antithrombin to restore coagulation balance, this innovation offers people living with hemophilia A or B, with or without inhibitors, a novel treatment option.”
Qfitlia leverages small-interfering RNA technology, enabling low-frequency, low-volume subcutaneous injections. It addresses a population of more than 40,000 hemophilia patients in China. Reported side effects include viral infections, nasopharyngitis, bacterial infections, and serious but rare events such as thrombosis and hepatotoxicity.
Similarly, Cablivi, a first-in-class Nanobody therapy, targets von Willebrand factor to prevent microthrombi formation, offering a new option for aTTP/iTTP, a rare, life-threatening clotting disorder with a mortality rate of up to 20% despite standard treatment. About 2,700 patients are diagnosed annually in China. The therapy is approved for adults and adolescents aged 12 and older, weighing at least 40 kg, and is used alongside plasma exchange and immunosuppressive therapy.
“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,” said Foard.
