Sanofi gets approval for 2 rare hematologic diseases medicines in China

Qfitlia is the antithrombin (AT)-lowering therapy for routine prophylaxis in people with hemophilia. Qfitlia is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in pediatric patients 12 years of age and older, and adults with severe hemophilia A (coagulation factor VIII deficiency, FVIII<1%) with or without factor VIII inhibitors, or severe hemophilia B (coagulation factor IX deficiency, FIX<1%) with or without factor IX inhibitors.

This approval is based on data from the ATLAS phase 3 studies that demonstrated clinically meaningful bleed protection as measured by annualized bleeding rates (ABR) across hemophilia patients with or without inhibitors. By lowering AT, a protein that inhibits blood clotting, Qfitlia helps increase thrombin generation to restore hemostasis in people with hemophilia. Qfitlia uses small-interfering RNA technology, which enables low treatment frequency, subcutaneous injections, and low-volume dosing. Hemophilia affects more than 40,000 people in China.

Cablivi is the Nanobody targeted therapy 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 approval brings an medicine specifically indicated for this rare and life-threatening blood clotting disorder to China, where approximately 2700 patients are diagnosed annually. Despite standard treatments, aTTP/iTTP carries a mortality rate of up to 20%. Cablivi targets von Willebrand factor (vWF), a protein in the blood involved in hemostasis, and is designed to inhibit the interaction between vWF and platelets. Used in conjunction with plasma exchange and immunosuppressive therapy, it helps by inhibiting the formation of microthrombi, which contribute to organ damage during the course of the disease.

These two approvals expand Sanofi's rare hematology portfolio in China, addressing critical unmet needs across both chronic bleeding disorders and acute clotting emergencies.

“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. “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.”

In the ATLAS clinical development program, Qfitlia demonstrated low bleed rates across subgroups with as few as six injections a year. Key results include:

• Significant bleed reduction by 71% in ABR for patients without inhibitors treated with Qfitlia prophylaxis compared to clotting factor concentrate on-demand (estimated mean: ABR 9.0 vs. 31.4, respectively; p<.0001) and by 73% in ABR compared to bypassing agent on-demand for patients with inhibitors (estimated mean: ABR 5.1 vs. 19.1, respectively; p<0.0006)
• Median observed ABR during the open-label extension study was 3.8 (interquartile range (IQR): 0.0–11.2) in patients without inhibitors and 1.9 (IQR: 0.0–5.6) in patients with inhibitors
• Nearly half of patients in the open-label extension study experienced one or fewer bleeds (47% 0-1 bleeds and 31% 0 bleeds)
• Nearly 80% of participants were on a regimen of six injections per year by the conclusion of the open-label extension study, and 94% achieved target AT levels with 0-1 dose adjustments

“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, China. “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.”