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Sanofi Lands Back-to-Back Rare Disease Approvals in China

MarketDash Editorial Team
13 hours ago
China's drug regulator just gave Sanofi the green light for two rare blood disorder treatments, marking the pharma giant's fourth and fifth approvals in the country this year as it builds out its hematology franchise.

Sanofi SA (SNY) just notched two major wins in China, securing regulatory approval for a pair of rare blood disorder treatments that could help thousands of patients while strengthening the French drugmaker's foothold in the world's second-largest pharmaceutical market.

China's National Medical Products Administration approved Qfitlia (fitusiran) for hemophilia and Cablivi (caplacizumab) for acquired thrombotic thrombocytopenic purpura, bringing Sanofi's China approval count to five for the year. The company previously landed approvals for Tzield in stage 2 type 1 diabetes and Sarclisa for multiple myeloma indications.

What These Drugs Actually Do

Qfitlia is approved for routine prophylaxis in patients 12 and older with severe hemophilia A, a condition affecting more than 40,000 people in China. The drug works by lowering antithrombin (AT), a protein that normally inhibits blood clotting. By reducing AT levels, Qfitlia boosts thrombin generation and helps restore normal blood clotting in hemophilia patients who otherwise bleed too easily.

The approval draws on data from the ATLAS phase 3 studies, which showed meaningful bleed protection across different patient groups. Here's where things get impressive: Qfitlia cut bleeding rates by 71% compared to on-demand clotting factor concentrate in patients without inhibitors, and by 73% compared to bypassing agents in patients with inhibitors. Many patients needed as few as six injections per year.

During the open-label extension study, the median annualized bleeding rate dropped to 3.8 in patients without inhibitors and just 1.9 in those with inhibitors. Nearly half of participants experienced one or fewer bleeds (47% had 0-1 bleeds, 31% had zero). By the study's conclusion, almost 80% of participants were on a six-injection-per-year regimen, and 94% hit target AT levels with minimal dose adjustments.

The Other Approval

Cablivi represents the first Nanobody-targeted therapy designed specifically for acquired thrombotic thrombocytopenic purpura in adults and adolescents 12 or older weighing at least 40 kg. About 2,700 patients receive this diagnosis annually in China.

This drug takes a different approach, targeting von Willebrand factor, a blood protein involved in clotting. Cablivi blocks the interaction between this protein and platelets, preventing the formation of microthrombi that damage organs during the disease's progression. It's used alongside plasma exchange and immunosuppressive therapy as part of a comprehensive treatment strategy.

Together, these approvals deepen Sanofi's rare hematology portfolio in China, addressing diseases that affect relatively small patient populations but create significant treatment challenges. The company is building momentum in the Chinese market across multiple therapeutic areas, from rare blood disorders to diabetes and cancer.

SNY Price Action: Sanofi stock traded up 1.31% at $48.89 Thursday.

Back to News

Sanofi Lands Back-to-Back Rare Disease Approvals in China

MarketDash Editorial Team
13 hours ago
China's drug regulator just gave Sanofi the green light for two rare blood disorder treatments, marking the pharma giant's fourth and fifth approvals in the country this year as it builds out its hematology franchise.

Sanofi SA (SNY) just notched two major wins in China, securing regulatory approval for a pair of rare blood disorder treatments that could help thousands of patients while strengthening the French drugmaker's foothold in the world's second-largest pharmaceutical market.

China's National Medical Products Administration approved Qfitlia (fitusiran) for hemophilia and Cablivi (caplacizumab) for acquired thrombotic thrombocytopenic purpura, bringing Sanofi's China approval count to five for the year. The company previously landed approvals for Tzield in stage 2 type 1 diabetes and Sarclisa for multiple myeloma indications.

What These Drugs Actually Do

Qfitlia is approved for routine prophylaxis in patients 12 and older with severe hemophilia A, a condition affecting more than 40,000 people in China. The drug works by lowering antithrombin (AT), a protein that normally inhibits blood clotting. By reducing AT levels, Qfitlia boosts thrombin generation and helps restore normal blood clotting in hemophilia patients who otherwise bleed too easily.

The approval draws on data from the ATLAS phase 3 studies, which showed meaningful bleed protection across different patient groups. Here's where things get impressive: Qfitlia cut bleeding rates by 71% compared to on-demand clotting factor concentrate in patients without inhibitors, and by 73% compared to bypassing agents in patients with inhibitors. Many patients needed as few as six injections per year.

During the open-label extension study, the median annualized bleeding rate dropped to 3.8 in patients without inhibitors and just 1.9 in those with inhibitors. Nearly half of participants experienced one or fewer bleeds (47% had 0-1 bleeds, 31% had zero). By the study's conclusion, almost 80% of participants were on a six-injection-per-year regimen, and 94% hit target AT levels with minimal dose adjustments.

The Other Approval

Cablivi represents the first Nanobody-targeted therapy designed specifically for acquired thrombotic thrombocytopenic purpura in adults and adolescents 12 or older weighing at least 40 kg. About 2,700 patients receive this diagnosis annually in China.

This drug takes a different approach, targeting von Willebrand factor, a blood protein involved in clotting. Cablivi blocks the interaction between this protein and platelets, preventing the formation of microthrombi that damage organs during the disease's progression. It's used alongside plasma exchange and immunosuppressive therapy as part of a comprehensive treatment strategy.

Together, these approvals deepen Sanofi's rare hematology portfolio in China, addressing diseases that affect relatively small patient populations but create significant treatment challenges. The company is building momentum in the Chinese market across multiple therapeutic areas, from rare blood disorders to diabetes and cancer.

SNY Price Action: Sanofi stock traded up 1.31% at $48.89 Thursday.